trials
Trials
Landmark and current trials that define the standard of care.
401 trials
| Products | Cancers | Sponsor | ||||
|---|---|---|---|---|---|---|
ASCERTAIN-V NCT04657081 ASCERTAIN-V is the study behind the first all-oral AML regimen, approved in May 2026. | Decitabine + cedazuridine, Venetoclax | Acute myeloid leukaemia | Taiho Oncology | 2026 | ||
AZUR-1 NCT05723770 AZUR-1 is the registrational trial of the 'no surgery, no radiation, no chemo' approach for mismatch-repair-deficient rectal cancer, built on the MSK study where every patient had a complete response. | Dostarlimab | Colorectal cancer | GSK | 2026 | ||
BL-B01D1-307 NCT06382142 The first phase 3 win for a bispecific ADC, in triple-negative breast cancer, announced February 2026. | Izalontamab brengitecan | Triple-negative breast cancer | SystImmune, BMS | 2026 | ||
CLARITY-Gastric 01 NCT06346392 The first ADC against Claudin 18.2 to prove it extends life, announced July 2026, giving stomach cancer its first ADC beyond HER2. | Sonesitatug vedotin, Ramucirumab | Gastric & gastro-oesophageal junction cancer | AstraZeneca | 2026 | ||
EMERALD-3 NCT05301842 In 2026 a dual-immunotherapy regimen plus lenvatinib added to TACE cut the risk of progression by about 30%; whether it extends life is not yet known. | Durvalumab, Tremelimumab, Lenvatinib | Hepatocellular carcinoma | AstraZeneca | 2026 | ||
FOURLIGHT-1 NCT06105632 In FOURLIGHT-1, a CDK4-only inhibitor designed to avoid the low blood counts of current CDK4/6 drugs improved progression-free survival in second line. | Atirmociclib | HR-positive / HER2-negative breast cancer | Pfizer | 2026 | ||
HERIZON-GEA-01 NCT05152147 A two-armed HER2 antibody beat Herceptin head-to-head as first-line treatment for HER2-positive stomach cancer, the first such win since 2010. | Zanidatamab, Trastuzumab, Tislelizumab | Gastric & gastro-oesophageal junction cancer, Oesophageal cancer | Jazz, BeOne | 2026 | ||
INSIGHT NCT05734105 A blood-test-selected trial testing whether matching the drug to the specific resistance mutation beats the standard second-line drug. | Ripretinib | Sarcomas | 2026 | |||
INTerpath-001 (V940-001) NCT05933577 INTerpath-001 was the first positive phase 3 trial of a personalised cancer vaccine, announced 19 August 2026. | Intismeran autogene, Pembrolizumab | Melanoma | Moderna, Merck | 2026 | ||
Krascendo 1 NCT06497556 The first head-to-head trial among KRAS drugs: Roche's divarasib beat the two approved pills on progression and survival. | Divarasib, Sotorasib, Adagrasib | Non-small-cell lung cancer | Roche | 2026 | ||
LITESPARK-022 NCT05239728 Adding the HIF-2α pill to a year of adjuvant pembrolizumab further reduced recurrence, and became the first adjuvant combination approved in kidney cancer. | Belzutifan, Pembrolizumab | Renal cell carcinoma | Merck | 2026 | ||
PANKU-Esophagus01 (BL-B01D1-305) NCT06304974 The first bispecific ADC to extend both progression-free and overall survival in oesophageal cancer, for patients whose immunotherapy has stopped working. | Izalontamab brengitecan | Oesophageal cancer | SystImmune, BMS | 2026 | ||
RASolute 302 NCT06625320 The trial that nearly doubled survival in previously treated pancreatic cancer, presented in the ASCO 2026 plenary. The biggest result in the disease's history. | Daraxonrasib | Pancreatic ductal adenocarcinoma | Revolution Medicines | 2026 | ||
ALASCCA NCT02647099 A Nordic trial showing that three years of low-dose aspirin roughly halves recurrence in bowel cancers carrying a particular set of mutations, a biomarker-directed use of a drug that costs pennies. | — | Colorectal cancer | 2025 | |||
ALKOVE-1 NCT05384626 The registrational study of a fourth-generation ALK pill designed to work after lorlatinib and to spare the brain-related side effects. | Neladalkib | Non-small-cell lung cancer | Nuvalent | 2025 | ||
ALPHAMEDIX-02 NCT05153772 An alpha-emitting radioligand met all its primary endpoints, with responses in more than half of patients new to radioligand therapy. | 212Pb-DOTAMTATE | Neuroendocrine tumours | RadioMedix, Orano Med, Sanofi | 2025 | ||
ASCENT-03 NCT05382299 Moved Trodelvy into first-line use for triple-negative patients who cannot receive immunotherapy. | Sacituzumab govitecan | Triple-negative breast cancer | Gilead | 2025 | ||
ASCENT-04 / KEYNOTE-D19 NCT05382286 Showed that pairing an ADC with immunotherapy beats chemotherapy plus immunotherapy in first-line PD-L1-positive TNBC. | Sacituzumab govitecan, Pembrolizumab | Triple-negative breast cancer | Gilead, Merck | 2025 | ||
ATOMIC (Alliance A021502) NCT02912559 Adding a year of immunotherapy to chemotherapy after surgery halved recurrences in stage III colon cancers with a broken DNA spell-checker. | Atezolizumab, FOLFOX | Colorectal cancer | Alliance, NCI | 2025 | ||
BREAKWATER NCT04607421 Doubled survival, from about 15 to about 30 months, in the worst-prognosis genetic subtype of bowel cancer by adding two targeted drugs to first-line chemotherapy. | Encorafenib, Cetuximab, FOLFOX, FOLFIRI | Colorectal cancer | Pfizer | 2025 | ||
CAPItello-281 NCT04493853 CAPItello-281 delivered the first AKT inhibitor success in prostate cancer, for the ~25% of men whose tumours have lost PTEN. | Capivasertib, Abiraterone acetate | Prostate cancer | AstraZeneca | 2025 | ||
CHALLENGE (CCTG CO.21) NCT00819208 The first randomised trial to show that a coached exercise programme after cancer treatment reduces recurrence and death, in colon cancer. | FOLFOX | Colorectal cancer | Canadian Cancer Trials Group | 2025 | ||
COMPETE NCT03049189 The first head-to-head trial of a radioligand against a targeted pill in neuroendocrine tumours; the radioligand won on progression-free survival. | 177Lu-edotreotide, Everolimus | Neuroendocrine tumours | ITM Isotope Technologies Munich | 2025 | ||
DeLLphi-304 NCT05740566 DeLLphi-304 was the first trial in which a T-cell engager improved survival in a common solid tumour. | Tarlatamab | Small-cell lung cancer | Amgen | 2025 | ||
DESTINY-Breast05 NCT04622319 Enhertu cut recurrence or death by more than half compared with Kadcyla in women with cancer left after pre-surgery treatment, replacing the KATHERINE standard. | Trastuzumab deruxtecan, Trastuzumab emtansine | HER2-positive breast cancer | Daiichi Sankyo, AstraZeneca | 2025 | ||
DESTINY-Breast09 NCT04784715 Showed Enhertu plus pertuzumab beats the decade-old first-line standard for HER2-positive metastatic breast cancer. | Trastuzumab deruxtecan | HER2-positive breast cancer | Daiichi Sankyo, AstraZeneca | 2025 | ||
DESTINY-Breast11 NCT05113251 DESTINY-Breast11 brought Enhertu into pre-surgery treatment of HER2-positive breast cancer, replacing anthracyclines. | Trastuzumab deruxtecan | HER2-positive breast cancer | Daiichi Sankyo, AstraZeneca | 2025 | ||
DESTINY-Gastric04 NCT04704934 The first randomised proof that a HER2 drug beats standard chemotherapy in second-line stomach cancer: Enhertu added about three months of life. | Trastuzumab deruxtecan, Ramucirumab | Gastric & gastro-oesophageal junction cancer | Daiichi Sankyo, AstraZeneca | 2025 | ||
ECLIPSE NCT05204927 Curium's PSMA radioligand met its progression endpoint; survival data and an FDA filing are pending. | 177Lu-PSMA-I&T | Prostate cancer | Curium | 2025 | ||
EV-303 / KEYNOTE-905 NCT03924895 For patients who cannot have cisplatin, the ADC-immunotherapy pair around surgery cut the risk of recurrence or death by 60% and the risk of death by half. | Enfortumab vedotin, Pembrolizumab | Bladder & urothelial cancer | Astellas, Pfizer, Merck | 2025 | ||
EV-304 / KEYNOTE-B15 NCT04700124 The trial that could replace chemotherapy before bladder removal in fit patients with an ADC plus immunotherapy. | Enfortumab vedotin, Pembrolizumab | Bladder & urothelial cancer | Merck, Astellas, Pfizer | 2025 | ||
evERA NCT05306340 In evERA, pairing an oral SERD with everolimus after CDK4/6 failure delayed progression by more than three months, and by four and a half months in ESR1-mutant tumours. | Giredestrant, Everolimus | HR-positive / HER2-negative breast cancer | Roche, Genentech | 2025 | ||
frontMIND NCT04824092 The first frontline regimen to beat R-CHOP in high-risk large B-cell lymphoma since rituximab, by adding a CD19 antibody and lenalidomide. | Tafasitamab, Lenalidomide | Diffuse large B-cell lymphoma | Incyte, MorphoSys | 2025 | ||
HER2CLIMB-05 NCT05132582 In HER2CLIMB-05, adding the brain-penetrant pill tucatinib to maintenance antibodies after first-line chemotherapy delayed progression by more than eight months. | Tucatinib, Trastuzumab, Pertuzumab | HER2-positive breast cancer | Pfizer | 2025 | ||
HORIZON-Breast01 NCT05424835 A Chinese HER2 ADC cut the risk of progression by nearly 80% versus a pill-plus-chemotherapy standard, matching the scale of Enhertu's results. | Trastuzumab rezetecan, Pyrotinib | HER2-positive breast cancer | Jiangsu Hengrui | 2025 | ||
IMforte NCT05091567 IMforte produced the first maintenance treatment ever approved for extensive-stage small-cell lung cancer, adding lurbinectedin to the immunotherapy that continues after chemotherapy. | Lurbinectedin, Atezolizumab | Small-cell lung cancer | Roche, Jazz, PharmaMar | 2025 | ||
iMMagine-1 NCT05396885 iMMagine-1 is the pivotal study behind anito-cel's BLA, with responses in nearly every patient. | Anitocabtagene autoleucel | Multiple myeloma | , Kite | 2025 | ||
IMvigor011 NCT04660344 The first trial to use a blood test for leftover cancer to decide who gets immunotherapy, and it worked. | Atezolizumab, Signatera | Bladder & urothelial cancer | Roche | 2025 | ||
IoN NCT01398085 The UK trial confirming that low-risk thyroid cancer patients can safely avoid radioactive iodine, published in 2025. | — | Thyroid cancer | Cancer Research UK, | 2025 | ||
KEYNOTE-689 NCT03765918 The first new treatment for curable head and neck cancer in six years: immunotherapy around surgery doubled the time patients stayed free of events. | Pembrolizumab | Head and neck squamous cell carcinoma | Merck | 2025 | ||
KEYNOTE-B96 / ENGOT-ov65 NCT05116189 After a decade of failures, the first immunotherapy trial to extend survival in ovarian cancer, leading to the first checkpoint-inhibitor approval in the disease. | Pembrolizumab, Paclitaxel / nab-paclitaxel, Bevacizumab | Ovarian cancer | Merck | 2025 | ||
KOMET-001 NCT04067336 KOMET-001 was the registration trial for ziftomenib: a quarter of heavily pretreated patients with NPM1-mutated AML reached complete remission on a once-daily pill. | Ziftomenib | Acute myeloid leukaemia | Kura Oncology | 2025 | ||
lidERA NCT04961996 The first oral SERD to reduce recurrence after surgery, by about 30%, compared with today's hormone pills. | Giredestrant | HR-positive / HER2-negative breast cancer | Roche, Genentech | 2025 | ||
MajesTEC-3 NCT05083169 A bispecific plus daratumumab cut the risk of progression by more than 80% versus standard doublets, bringing bispecifics to second line. | Teclistamab, Daratumumab | Multiple myeloma | Janssen | 2025 | ||
MATTERHORN NCT04592913 Adding immunotherapy before and after surgery cut deaths in early stomach cancer; nearly seven in ten patients were alive at three years. | Durvalumab, FLOT | Gastric & gastro-oesophageal junction cancer | AstraZeneca | 2025 | ||
PANOVA-3 NCT03377491 PANOVA-3 is the trial behind the 2026 approval of a wearable electric-field device for pancreatic cancer, the first new approval in locally advanced disease in decades. | Optune / Optune Pax, Gemcitabine + nab-paclitaxel | Pancreatic ductal adenocarcinoma | Novocure | 2025 | ||
POTOMAC NCT03528694 Adding a year of durvalumab to standard BCG bladder instillations cut the risk of recurrence or progression by about a third, the first systemic immunotherapy approved in early bladder cancer. | Durvalumab, Intravesical BCG | Bladder & urothelial cancer | AstraZeneca | 2025 | ||
PSMAddition NCT04720157 Moved Pluvicto to the very first treatment of metastatic prostate cancer; FDA approved this use on 31 July 2026. | Lutetium-177 vipivotide tetraxetan | Prostate cancer | Novartis | 2025 | ||
ROSELLA / GOG-3073 NCT05257408 Blocking cortisol signalling in tumour cells made chemotherapy work better and extended life in resistant ovarian cancer. | Relacorilant, Paclitaxel / nab-paclitaxel | Ovarian cancer | 2025 | |||
SERENA-6 NCT04964934 The first trial to change treatment because of a blood test rather than a scan: switching to camizestrant when an ESR1 mutation appeared in the blood delayed progression by seven months. | Camizestrant | HR-positive / HER2-negative breast cancer | AstraZeneca | 2025 | ||
SOHO-01 NCT05099172 SOHO-01 is the study behind the second oral HER2 pill for lung cancer, approved in November 2025. | Sevabertinib | Non-small-cell lung cancer | Bayer | 2025 | ||
SUNMO NCT05171647 A chemotherapy-free bispecific plus ADC doublet beat salvage chemotherapy in relapsed large B-cell lymphoma. | Mosunetuzumab, Polatuzumab vedotin | Diffuse large B-cell lymphoma | Roche | 2025 | ||
TROPION-Breast02 NCT05374512 The first trial to show an overall survival benefit for a first-line ADC in triple-negative breast cancer. | Datopotamab deruxtecan | Triple-negative breast cancer | AstraZeneca, Daiichi Sankyo | 2025 | ||
ACE-Breast-02 NCT04829604 A site-specifically conjugated HER2 ADC beat lapatinib-capecitabine on progression-free survival in China. | ARX788, Lapatinib | HER2-positive breast cancer | 2024 | |||
ADRIATIC NCT03703297 ADRIATIC brought the first improvement in curative-intent small-cell lung cancer treatment in 30 years: a year or two of immunotherapy after chemoradiation lengthens life. | Durvalumab | Small-cell lung cancer | AstraZeneca | 2024 | ||
AMPLIFY NCT03836261 AMPLIFY is the trial behind the first all-oral, fixed-duration CLL regimen approved in the US (February 2026): 14 cycles of two pills, then stop. | Acalabrutinib, Venetoclax, Obinutuzumab | Chronic lymphocytic leukaemia | AstraZeneca | 2024 | ||
AUGMENT-101 NCT04065399 The trial that turned menin inhibition from an idea into the first approved drug for KMT2A-rearranged leukaemia. | Revumenib | Acute myeloid leukaemia, Acute lymphoblastic leukaemia | 2024 | |||
BOND-003 NCT04452591 A cancer-killing virus instilled into the bladder produced complete responses in three quarters of patients whose BCG had failed, with almost no serious side effects. | Cretostimogene grenadenorepvec | Bladder & urothelial cancer | CG Oncology | 2024 | ||
BRUIN CLL-321 NCT04666038 The randomised trial that confirmed pirtobrutinib works after other BTK inhibitors fail, leading to full approval in December 2025. | Pirtobrutinib | Chronic lymphocytic leukaemia | Eli Lilly, Loxo | 2024 | ||
CABINET (Alliance A021602) NCT03375320 Cabozantinib tripled the time without progression in neuroendocrine tumours that had outgrown other treatments, leading to a 2025 approval. | Cabozantinib | Neuroendocrine tumours | Alliance, NCI, Exelixis | 2024 | ||
CEPHEUS NCT03652064 CEPHEUS extended the quadruplet advantage to patients who do not have a transplant. | Daratumumab, Bortezomib, Lenalidomide | Multiple myeloma | Janssen | 2024 | ||
CheckMate 8HW NCT04008030 Showed that a two-drug immunotherapy combination controls mismatch-repair-deficient bowel cancer for over four years on average, and beats immunotherapy alone. | Nivolumab, Ipilimumab | Colorectal cancer | BMS | 2024 | ||
CheckMate 9DW NCT04039607 Dual immune checkpoint blockade gave the longest median survival yet seen in a first-line liver cancer trial. | Nivolumab, Ipilimumab | Hepatocellular carcinoma | BMS | 2024 | ||
COG AALL1731 NCT03914625 In children with average-risk leukaemia, adding blinatumomab pushed three-year disease-free survival from 88% to 96%, one of the largest gains in decades. | Blinatumomab | Acute lymphoblastic leukaemia | Children's Oncology Group, NCI | 2024 | ||
COMPASSION-16 / AK104-303 NCT04982237 A two-headed antibody that blocks two immune brakes at once extended survival in advanced cervical cancer regardless of PD-L1 status. | Cadonilimab, Bevacizumab, Cisplatin, Paclitaxel / nab-paclitaxel | Cervical cancer | Akeso | 2024 | ||
DESTINY-Breast06 NCT04494425 Moved Enhertu ahead of chemotherapy in hormone-positive breast cancer and extended it to 'ultralow' HER2. | Trastuzumab deruxtecan | HR-positive / HER2-negative breast cancer | Daiichi Sankyo, AstraZeneca | 2024 | ||
DESTINY-Breast12 NCT04739761 Enhertu shrank brain metastases in most patients, including those with untreated lesions, answering a question its earlier trials had excluded. | Trastuzumab deruxtecan | HER2-positive breast cancer | AstraZeneca, Daiichi Sankyo | 2024 | ||
DREAMM-7 NCT04246047 The trial that brought the withdrawn myeloma ADC back, beating a daratumumab-based standard on progression and survival. | Belantamab mafodotin, Bortezomib | Multiple myeloma | GSK | 2024 | ||
DREAMM-8 NCT04484623 DREAMM-8 is the second confirmatory win for belantamab, in lenalidomide-exposed patients. | Belantamab mafodotin | Multiple myeloma | GSK | 2024 | ||
ECOG-ACRIN E1910 NCT02003222 Proved that adding the immunotherapy blinatumomab to standard treatment saves lives even in patients whose leukaemia was already undetectable. | Blinatumomab | Acute lymphoblastic leukaemia | ECOG-ACRIN, NCI | 2024 | ||
EMBER-3 NCT04975308 An oral SERD that works alone in ESR1-mutant tumours and, combined with abemaciclib, doubles progression-free time in everyone regardless of mutation. | Imlunestrant, Abemaciclib | HR-positive / HER2-negative breast cancer | Eli Lilly | 2024 | ||
FELIX NCT04404660 The trial that showed a gentler CAR-T design could treat adult ALL with a fraction of the severe side effects. | Obecabtagene autoleucel | Acute lymphoblastic leukaemia | 2024 | |||
GHSG HD21 NCT02661503 A new brentuximab-based intensive regimen matched Europe's most effective (and most toxic) chemotherapy with far fewer side effects. | Brentuximab vedotin, Doxorubicin | Hodgkin lymphoma | German Hodgkin Study Group | 2024 | ||
HARMONi-2 NCT05499390 The first head-to-head trial in which a new drug beat Keytruda, in China; a survival benefit followed in 2026. | Ivonescimab, Pembrolizumab | Non-small-cell lung cancer | Akeso | 2024 | ||
IGNYTE-ESO NCT03967223 IGNYTE-ESO was the pivotal study for the second sarcoma TCR-T, with responses in 42% of patients with two rare sarcomas. | Letetresgene autoleucel | Sarcomas | Adaptimmune, GSK | 2024 | ||
IMROZ NCT03319667 In IMROZ, isatuximab added to VRd cut progression by 40% in older patients not having a transplant. | Isatuximab, Bortezomib, Lenalidomide | Multiple myeloma | Sanofi | 2024 | ||
KRYSTAL-12 NCT04685135 Confirmed that the KRAS pill adagrasib beats chemotherapy after first-line treatment, though the gain is modest. | Adagrasib | Non-small-cell lung cancer | BMS (Mirati) | 2024 | ||
LAURA NCT03521154 For stage III lung cancers with an EGFR mutation, a targeted pill after chemoradiation cut progression by more than 80%. | Osimertinib | Non-small-cell lung cancer | AstraZeneca | 2024 | ||
LINKER-MM1 NCT03761108 LINKER-MM1 is the pivotal study for linvoseltamab, with 45% complete responses. | Linvoseltamab | Multiple myeloma | Regeneron | 2024 | ||
MOTION NCT05059262 Vimseltinib shrank tenosynovial giant cell tumours in 40% of patients versus none on placebo, with better joint function. | Vimseltinib | Sarcomas | 2024 | |||
NADINA NCT04949113 Giving the immunotherapy doublet before surgery, instead of nivolumab after, cut the risk of recurrence by about two thirds and let most patients skip further treatment. | Nivolumab, Ipilimumab | Melanoma | Netherlands Cancer Institute, | 2024 | ||
NIAGARA NCT03732677 The first immunotherapy shown to improve survival when given around bladder-removal surgery. | Durvalumab | Bladder & urothelial cancer | AstraZeneca | 2024 | ||
PhALLCON NCT03589326 Head to head, the third-generation TKI ponatinib doubled the rate of deep, undetectable remission over imatinib in newly diagnosed Ph-positive ALL. | Ponatinib, Imatinib | Acute lymphoblastic leukaemia | Takeda | 2024 | ||
PHERGain NCT03161353 Using an early PET scan to spot responders let a third of women be cured of HER2-positive breast cancer without any chemotherapy. | Trastuzumab, Pertuzumab | HER2-positive breast cancer | 2024 | |||
Ponsegromab phase 2 in cancer cachexia NCT05546476 The first drug to hit the hormone behind cancer wasting: patients on the highest dose gained nearly 3 kg more than placebo in 12 weeks and reported better appetite and more activity. | — | Non-small-cell lung cancer, Pancreatic ductal adenocarcinoma, Colorectal cancer | Pfizer | 2024 | ||
postMONARCH NCT05169567 Continuing CDK4/6 blockade with a different drug after the first one fails gives a small but real benefit. | Abemaciclib | HR-positive / HER2-negative breast cancer | Eli Lilly | 2024 | ||
RAMP 201 NCT04625270 RAMP 201 produced the first approved treatment designed for low-grade serous ovarian cancer, a slow-growing type driven by the RAS pathway that shrugs off chemotherapy. | Avutometinib + defactinib | Ovarian cancer | 2024 | |||
SunRISe-1 NCT04640623 A tiny pretzel-shaped device that slowly releases chemotherapy inside the bladder cleared carcinoma in situ in over 80% of patients whose BCG had failed. | Gemcitabine intravesical system | Bladder & urothelial cancer | Johnson & Johnson | 2024 | ||
ALINA NCT03456076 Showed that giving an ALK pill after surgery, instead of chemotherapy, sharply reduces recurrence, including in the brain. | Alectinib | Non-small-cell lung cancer | Roche | 2023 | ||
BEATcc / ENGOT-Cx10 / GOG-3030 NCT03556839 A second immunotherapy confirmed that adding a checkpoint inhibitor to chemotherapy plus bevacizumab extends life in advanced cervical cancer. | Atezolizumab, Bevacizumab, Cisplatin, Paclitaxel / nab-paclitaxel | Cervical cancer | , Roche | 2023 | ||
CARES-310 NCT03764293 A PD-1 antibody plus an oral anti-VEGF pill beat sorafenib on survival, one of the largest benefits seen in liver cancer. | Camrelizumab + rivoceranib | Hepatocellular carcinoma | Jiangsu Hengrui, | 2023 | ||
CARTITUDE-4 NCT04181827 The first randomised trial to show a CAR-T improves survival in myeloma, as early as second line. | Ciltacabtagene autoleucel | Multiple myeloma | Janssen, Legend | 2023 | ||
CLL13 / GAIA NCT02950051 In fit patients, one year of venetoclax plus obinutuzumab (with or without ibrutinib) clearly beat the old chemotherapy standard. | Venetoclax, Obinutuzumab, Rituximab, Ibrutinib | Chronic lymphocytic leukaemia | 2023 | |||
CodeBreaK 300 NCT05198934 CodeBreaK 300 showed that a KRAS drug needs an EGFR antibody partner to work in colorectal cancer. | Sotorasib | Colorectal cancer | Amgen | 2023 | ||
DESTINY-CRC02 NCT04744831 Enhertu shrank about four in ten heavily pretreated HER2-positive bowel cancers, including ones that had already stopped responding to other HER2 drugs. | Trastuzumab deruxtecan | Colorectal cancer | Daiichi Sankyo, AstraZeneca | 2023 | ||
DESTINY-PanTumor02 NCT04482309 Enhertu shrank tumours across many HER2-positive cancers, with endometrial cancer among the best responders, leading to the first tumour-agnostic HER2 approval. | Trastuzumab deruxtecan | Endometrial cancer, Cervical cancer | AstraZeneca, Daiichi Sankyo | 2023 | ||
DUO-E / GOG-3041 / ENGOT-EN10 NCT04269200 A third immunotherapy confirmed the benefit in dMMR disease and hinted that adding olaparib helps the mismatch-repair-proficient majority. | Durvalumab, Olaparib, Carboplatin, Paclitaxel / nab-paclitaxel | Endometrial cancer | AstraZeneca | 2023 | ||
EMBARK NCT02319837 Showed that treating a fast-rising PSA after surgery or radiation with enzalutamide delays spread. | Enzalutamide | Prostate cancer | Pfizer, Astellas | 2023 | ||
EV-302 / KEYNOTE-A39 NCT04223856 Nearly doubled survival in advanced bladder cancer, ending 40 years of platinum chemotherapy as the standard. | Enfortumab vedotin, Pembrolizumab | Bladder & urothelial cancer | Astellas, Pfizer, Merck | 2023 | ||
FLAURA2 NCT04035486 Adding chemotherapy to osimertinib extended both progression-free and, in 2025, overall survival. | Osimertinib | Non-small-cell lung cancer | AstraZeneca | 2023 | ||
FRESCO-2 NCT04322539 A selective VEGF-receptor pill gave a modest but real survival gain in patients with no remaining standard treatment. | Fruquintinib | Colorectal cancer | , Takeda | 2023 | ||
GD2-CART01 (Bambino Gesù phase 1/2) NCT03373097 GD2-CART01 was the first CAR-T to produce durable complete remissions in a childhood solid tumour: two-thirds responded and a third achieved complete remission. | — | Neuroblastoma | 2023 | |||
INAVO120 NCT04191499 A triple combination that doubled progression-free time and, unusually for this disease, extended survival by seven months in a hard-to-treat group. | Inavolisib, Palbociclib | HR-positive / HER2-negative breast cancer | Roche, Genentech | 2023 | ||
IND.227 / KEYNOTE-483 NCT02784171 Showed that adding a PD-1 blocker to standard chemotherapy helps in mesothelioma, giving a second immunotherapy-based first-line option. | Pembrolizumab, Pemetrexed | Mesothelioma | Canadian Cancer Trials Group, Merck | 2023 | ||
INDIGO NCT04164901 The first targeted-therapy win in low-grade brain tumours: a pill that more than doubled the time before the tumour grew, delaying radiation and chemotherapy by years. | Vorasidenib | Glioma & glioblastoma | Servier | 2023 | ||
innovaTV 301 / ENGOT-cx12 / GOG-3057 NCT04697628 The first ADC to extend life in cervical cancer, for women whose disease has come back after chemotherapy. | Tisotumab vedotin | Cervical cancer | Genmab, Pfizer (Seagen) | 2023 | ||
INTERLACE NCT01566240 Six weeks of cheap, generic chemotherapy before standard chemoradiation cut deaths by 40%, an advance usable anywhere in the world. | Carboplatin, Paclitaxel / nab-paclitaxel, Cisplatin | Cervical cancer | , CRUK | 2023 | ||
KarMMa-3 NCT03651128 Ide-cel tripled progression-free time versus standard regimens in heavily pretreated myeloma, but did not clearly extend life after most control patients crossed over. | Idecabtagene vicleucel | Multiple myeloma | BMS, | 2023 | ||
KEYNOTE-671 NCT03425643 Showed that immunotherapy given both before and after lung cancer surgery lengthens survival. | Pembrolizumab | Non-small-cell lung cancer | Merck | 2023 | ||
KEYNOTE-859 NCT03675737 Confirmed that adding a PD-1 blocker to first-line chemotherapy helps in stomach cancer, with the biggest gain in tumours rich in PD-L1. | Pembrolizumab | Gastric & gastro-oesophageal junction cancer | Merck | 2023 | ||
KEYNOTE-966 NCT04003636 A second immunotherapy trial confirmed the modest survival benefit of adding PD-1 blockade to chemotherapy in bile duct cancer. | Pembrolizumab, Gemcitabine + cisplatin | Biliary tract cancer | Merck | 2023 | ||
KEYNOTE-A18 / ENGOT-cx11 / GOG-3047 NCT04221945 Adding immunotherapy to curative chemoradiation for locally advanced cervical cancer improved both control and survival, the first such advance in two decades. | Pembrolizumab | Cervical cancer | Merck | 2023 | ||
LIBRETTO-431 NCT04194944 Proved that a RET pill is better than chemotherapy with or without immunotherapy as first treatment for RET-fusion lung cancer. | Selpercatinib | Non-small-cell lung cancer | Eli Lilly | 2023 | ||
LIBRETTO-531 NCT04211337 Showed that a drug built specifically for the RET mutation beats the older multi-target pills in medullary thyroid cancer, with far fewer side effects. | Selpercatinib | Thyroid cancer | Eli Lilly | 2023 | ||
LITESPARK-005 NCT04195750 The HIF-2α inhibitor beat the old standard everolimus after immunotherapy and targeted therapy had failed, with durable responses in a subset. | Belzutifan, Everolimus | Renal cell carcinoma | Merck | 2023 | ||
Low-dose olanzapine for cancer anorexia (Tata Memorial) A randomised trial in India of a 2.5 mg dose of a decades-old, very cheap tablet. Six in ten patients gained more than 5% of their weight, against one in ten on placebo. | — | Gastric & gastro-oesophageal junction cancer, Non-small-cell lung cancer, Pancreatic ductal adenocarcinoma, Hepatocellular carcinoma | Tata Memorial Centre | 2023 | ||
| Elranatamab | Multiple myeloma | Pfizer | 2023 | |||
MARIPOSA NCT04487080 The first regimen to beat osimertinib in EGFR-mutant lung cancer, with a survival benefit exceeding a year. | Amivantamab | Non-small-cell lung cancer | Johnson & Johnson | 2023 | ||
MIRASOL / GOG-3045 NCT04209855 The first ADC to extend life in ovarian cancer, for the roughly one-third of tumours with high folate receptor alpha. | Mirvetuximab soravtansine | Ovarian cancer | ImmunoGen (AbbVie) | 2023 | ||
NAPOLI 3 NCT04083235 The first head-to-head trial of the two chemotherapy backbones, won narrowly by the four-drug regimen. | NALIRIFOX, Gemcitabine + nab-paclitaxel | Pancreatic ductal adenocarcinoma | Ipsen | 2023 | ||
NATALEE NCT03701334 Extended CDK4/6 inhibitors to a broad group of early hormone-positive breast cancer patients, including node-negative. | Ribociclib | HR-positive / HER2-negative breast cancer | Novartis | 2023 | ||
NMTRC003/003B (DFMO maintenance) NCT02395666 NMTRC003 was the single-arm study, compared against historical patients, that got eflornithine approved as maintenance; the design remains debated. | Eflornithine | Neuroblastoma | 2023 | |||
NRG-GY018 / KEYNOTE-868 NCT03914612 NRG-GY018 is the pembrolizumab twin of RUBY: immunotherapy plus chemotherapy cut the risk of progression by 70% in dMMR tumours and 46% in the rest. | Pembrolizumab, Carboplatin, Paclitaxel / nab-paclitaxel | Endometrial cancer | NRG Oncology, Merck | 2023 | ||
PERSEUS NCT03710603 Adding daratumumab to the standard three-drug induction and to maintenance cut the risk of progression by more than half in transplant-eligible myeloma. | Daratumumab, Bortezomib, Lenalidomide | Multiple myeloma | , Janssen | 2023 | ||
PREHAB: multimodal prehabilitation before colorectal cancer surgery PREHAB showed that four weeks of supervised training, protein and support before bowel cancer surgery cut severe complications and sped recovery, in an international randomised trial of 251 patients. | — | Colorectal cancer | 2023 | |||
| Lutetium-177 vipivotide tetraxetan | Prostate cancer | Novartis | 2023 | |||
RUBY / ENGOT-EN6 / GOG-3031 NCT03981796 Adding immunotherapy to first chemotherapy for advanced endometrial cancer extended life by more than a year on average, most dramatically in tumours with a broken DNA spell-checker. | Dostarlimab, Carboplatin, Paclitaxel / nab-paclitaxel | Endometrial cancer | GSK | 2023 | ||
SANO NCT05953181 For the third of patients whose tumour vanishes after chemoradiation, watching closely and operating only if it comes back gave the same survival as immediate surgery. | — | Oesophageal cancer | 2023 | |||
SPOTLIGHT & GLOW NCT03504397 Two trials that made Claudin 18.2 the third biomarker in stomach cancer, adding about two to three months of survival with an antibody against it. | Zolbetuximab, FOLFOX, CAPOX | Gastric & gastro-oesophageal junction cancer | Astellas | 2023 | ||
SUNLIGHT NCT04737187 Adding the old blood-vessel antibody to a late-line chemotherapy pill extended survival by three months in patients who had exhausted standard options. | Trifluridine/tipiracil, Bevacizumab | Colorectal cancer | , Servier | 2023 | ||
SWOG S1826 NCT03907488 Immunotherapy plus chemotherapy beat the previous best regimen with far less nerve damage, in the first Hodgkin trial to enrol children and adults together; approved March 2026. | Nivolumab, Brentuximab vedotin, Doxorubicin | Hodgkin lymphoma | SWOG, NCI (with COG) | 2023 | ||
TALAPRO-2 NCT03395197 The PARP-plus-hormone combination that eventually showed an overall survival benefit, in 2024-25. | Talazoparib, Enzalutamide | Prostate cancer | Pfizer | 2023 | ||
THOR NCT03390504 The first targeted pill to lengthen survival in bladder cancer, for the fifth of tumours with FGFR alterations. | Erdafitinib | Bladder & urothelial cancer | Johnson & Johnson | 2023 | ||
TRANSCEND CLL 004 NCT03331198 The study that brought CAR-T to CLL: one in five heavily pretreated patients achieved complete remission, most of them lasting. | Lisocabtagene maraleucel | Chronic lymphocytic leukaemia | BMS, | 2023 | ||
TROPION-Lung05 NCT04484142 The study that got Datroway approved for EGFR-mutant lung cancer after other treatments fail. | Datopotamab deruxtecan | Non-small-cell lung cancer | AstraZeneca, Daiichi Sankyo | 2023 | ||
AGILE NCT03173248 In IDH1-mutated AML, adding ivosidenib to azacitidine tripled survival, one of the largest effects ever seen in a randomised AML trial. | Ivosidenib, Azacitidine | Acute myeloid leukaemia | , Servier | 2022 | ||
ALPINE NCT03734016 The first head-to-head trial in which a newer BTK inhibitor was both safer and more effective than ibrutinib. | Zanubrutinib, Ibrutinib | Chronic lymphocytic leukaemia | BeOne (BeiGene) | 2022 | ||
ARASENS NCT02799602 Proved 'triplet therapy': adding darolutamide to hormone therapy plus chemotherapy reduces death by a third. | Darolutamide, Docetaxel | Prostate cancer | Bayer, | 2022 | ||
ASTRUM-005 NCT04063163 In ASTRUM-005, a Chinese PD-1 antibody produced the longest first-line survival of the chemo-immunotherapy trials; it is now approved in Europe and the UK but not yet in the US. | Serplulimab, Platinum + etoposide | Small-cell lung cancer | 2022 | |||
ATHENA-MONO / GOG-3020 NCT03522246 A third PARP inhibitor showed the same pattern: longer disease control after first-line chemotherapy, biggest in tumours with faulty DNA repair. | Rucaparib | Ovarian cancer | 2022 | |||
CAPItello-291 NCT04305496 Adding the AKT inhibitor capivasertib to fulvestrant doubled progression-free time, especially in tumours with PI3K-pathway mutations. | Capivasertib | HR-positive / HER2-negative breast cancer | AstraZeneca | 2022 | ||
CheckMate 816 NCT02998528 CheckMate 816 was the first trial to show that immunotherapy before lung cancer surgery improves survival. | Nivolumab | Non-small-cell lung cancer | BMS | 2022 | ||
CodeBreaK 200 NCT04303780 The first randomised trial of a KRAS drug: better than chemotherapy on progression, not on survival. | Sotorasib | Non-small-cell lung cancer | Amgen | 2022 | ||
DeFi NCT03785964 The first randomised trial to show a drug controls desmoid tumours, with better pain and function. | Nirogacestat | Sarcomas | 2022 | |||
DESTINY-Breast04 NCT03734029 Created a new category of breast cancer, HER2-low, by showing Enhertu works in tumours previously called HER2-negative. | Trastuzumab deruxtecan | HR-positive / HER2-negative breast cancer, Triple-negative breast cancer | Daiichi Sankyo, AstraZeneca | 2022 | ||
DYNAMIC ACTRN12615000381583 Showed that a blood test can safely halve the number of colon cancer patients given chemotherapy after surgery. | — | Colorectal cancer | 2022 | |||
EPCORE NHL-1 NCT03625037 EPCORE NHL-1 was the pivotal single-arm study behind epcoritamab's approval; about half of complete responders are still in remission at three years. | Epcoritamab | Diffuse large B-cell lymphoma | Genmab, AbbVie | 2022 | ||
ESTIMABL2 NCT01837745 Proved that most people with small, low-risk thyroid cancers can skip radioactive iodine after surgery without any increase in recurrence. | — | Thyroid cancer | Gustave Roussy, | 2022 | ||
FOENIX-CCA2 NCT02052778 Futibatinib produced responses in over 40% of patients whose bile duct cancer carried an FGFR2 fusion. | Futibatinib | Biliary tract cancer | 2022 | |||
HIMALAYA NCT03298451 A single dose of a CTLA-4 antibody added to PD-L1 blockade doubled five-year survival in liver cancer without needing an anti-VEGF drug. | Durvalumab, Tremelimumab | Hepatocellular carcinoma | AstraZeneca | 2022 | ||
KEN SHE (single-dose HPV vaccine) NCT03675256 One shot of HPV vaccine was 97.5% effective against the two most dangerous HPV types, making it realistic to vaccinate the whole world. | Nonavalent HPV vaccine | Cervical cancer, Head and neck squamous cell carcinoma | 2022 | |||
MajesTEC-1 NCT04557098 MajesTEC-1 was the pivotal single-arm study for the first myeloma bispecific, with responses in 63% of patients who had exhausted the main drug classes. | Teclistamab | Multiple myeloma | Janssen | 2022 | ||
MonumenTAL-1 NCT04634552 Showed that a second target, GPRC5D, works in myeloma, including after BCMA therapies have failed. | Talquetamab | Multiple myeloma | Janssen | 2022 | ||
NICHE-2 NCT03026140 In NICHE-2, four weeks of immunotherapy before surgery wiped out most mismatch-repair-deficient colon cancers, and nobody had relapsed three years later. | Nivolumab, Ipilimumab | Colorectal cancer | Netherlands Cancer Institute | 2022 | ||
PARADIGM NCT02394795 Proved that for RAS-normal tumours starting on the left side of the colon, an EGFR antibody beats the VEGF antibody as first partner for chemotherapy. | Panitumumab, Bevacizumab, FOLFOX | Colorectal cancer | Takeda (Japan) | 2022 | ||
PROpel NCT03732820 Showed PARP inhibitor plus abiraterone delays progression in first-line mCRPC, with the largest benefit in BRCA-mutant men. | Olaparib, Abiraterone acetate | Prostate cancer | AstraZeneca, Merck | 2022 | ||
QuANTUM-First NCT02668653 Roughly doubled median survival in the most aggressive FLT3 subtype by adding a selective FLT3 blocker to chemotherapy, including in patients over 60. | Quizartinib, Cytarabine + anthracycline | Acute myeloid leukaemia | Daiichi Sankyo | 2022 | ||
QUILT 3.032 NCT03022825 An IL-15 booster given with BCG produced complete responses in about 70% of patients with BCG-unresponsive carcinoma in situ and kept most bladders intact for years. | Nogapendekin alfa inbakicept, Intravesical BCG | Bladder & urothelial cancer | ImmunityBio | 2022 | ||
RATIONALE-306 NCT03783442 RATIONALE-306 is the trial behind tislelizumab's US approval for squamous oesophageal cancer, with the biggest gains in PD-L1-positive tumours. | Tislelizumab | Oesophageal cancer | BeOne (BeiGene) | 2022 | ||
RELATIVITY-047 NCT03470922 Proved that adding a LAG-3 blocker to PD-1 blockade delays progression, with far less toxicity than the ipilimumab combination. | Relatlimab + nivolumab, Nivolumab | Melanoma | BMS | 2022 | ||
SEQUOIA NCT03336333 Zanubrutinib beat chemoimmunotherapy in untreated CLL and delivered a 72% five-year progression-free rate in the hardest genetic subgroup. | Zanubrutinib, Venetoclax, Rituximab | Chronic lymphocytic leukaemia | BeOne (BeiGene) | 2022 | ||
SWOG S1801 NCT03698019 Simply moving three doses of the same drug to before surgery improved outcomes, a result that changed how the field thinks about timing. | Pembrolizumab | Melanoma | SWOG, NCI | 2022 | ||
TOPAZ-1 NCT03875235 TOPAZ-1 was the first immunotherapy success in bile duct cancer, with a small median gain but a growing tail of long survivors. | Durvalumab, Gemcitabine + cisplatin | Biliary tract cancer | AstraZeneca | 2022 | ||
TRANSFORM NCT03575351 The second trial to show a CAR-T beats transplant in early-relapsing large B-cell lymphoma. | — | Diffuse large B-cell lymphoma | BMS | 2022 | ||
TROPiCS-02 NCT03901339 The TROP2 ADC extended survival by about three months in heavily pretreated hormone-positive breast cancer. | Sacituzumab govitecan | HR-positive / HER2-negative breast cancer | Gilead | 2022 | ||
ARROW (thyroid cohorts) NCT03037385 ARROW is the single-arm study behind the second RET inhibitor's thyroid approvals. | Pralsetinib | Thyroid cancer | 2021 | |||
C-144-01 NCT02360579 The single-arm study that got the first cell therapy for a solid tumour approved: one in three patients responded after everything else had failed. | Lifileucel | Melanoma | 2021 | |||
CAPTIVATE NCT02910583 Fifteen months of two pills, then stop: three-quarters of patients had no detectable leukaemia in the blood, and most remained in remission years later. | Ibrutinib, Venetoclax | Chronic lymphocytic leukaemia | , AbbVie | 2021 | ||
CARTITUDE-1 NCT03548207 The study that showed one CAR-T infusion could keep a third of heavily pretreated myeloma patients progression-free for five years without further treatment. | Ciltacabtagene autoleucel | Multiple myeloma | Janssen, Legend | 2021 | ||
CheckMate 274 NCT02632409 A year of nivolumab after bladder or upper-tract surgery reduces recurrence and, with long follow-up, extends life. | Nivolumab | Bladder & urothelial cancer | BMS | 2021 | ||
CheckMate 648 NCT03143153 Showed two immunotherapy options for squamous oesophageal cancer, including one with no chemotherapy at all, both extending survival. | Nivolumab, Ipilimumab | Oesophageal cancer | BMS | 2021 | ||
CLEAR (KEYNOTE-581) NCT02811861 The combination with the longest progression-free survival ever reported in first-line kidney cancer, at nearly two years. | Lenvatinib, Pembrolizumab, Sunitinib, Everolimus | Renal cell carcinoma | Eisai, Merck | 2021 | ||
DESTINY-Breast03 NCT03529110 The head-to-head ADC trial where Enhertu beat Kadcyla by a wide margin, showing that payload and bystander effect matter. | Trastuzumab deruxtecan, Trastuzumab emtansine | HER2-positive breast cancer | Daiichi Sankyo, AstraZeneca | 2021 | ||
DREAMseq (ECOG-ACRIN EA6134) NCT02224781 Settled the order question: for BRAF-mutant melanoma, start with immunotherapy and save the targeted pills for later. | Nivolumab, Ipilimumab, Dabrafenib + trametinib | Melanoma | ECOG-ACRIN, NCI | 2021 | ||
EMERALD NCT03778931 The first oral oestrogen-receptor degrader to beat standard hormone therapy, with the benefit concentrated in tumours carrying ESR1 mutations. | Elacestrant | HR-positive / HER2-negative breast cancer | , Menarini | 2021 | ||
EMPOWER-Cervical 1 / GOG-3016 / ENGOT-cx9 NCT03257267 The first immunotherapy to extend life in recurrent cervical cancer, approved in Europe but declined by the FDA. | Cemiplimab | Cervical cancer | Regeneron, Sanofi | 2021 | ||
ESCORT-1st NCT03691090 ESCORT-1st is China's first-line immunotherapy trial for squamous oesophageal cancer, one of five that together made chemo-immunotherapy the global standard. | Camrelizumab | Oesophageal cancer | Jiangsu Hengrui | 2021 | ||
GLOW NCT03462719 Fifteen months of two oral drugs cut the risk of progression by nearly 80% versus chemoimmunotherapy in older patients, and later showed a survival advantage. | Ibrutinib, Venetoclax, Obinutuzumab | Chronic lymphocytic leukaemia | Janssen | 2021 | ||
IMCgp100-202 NCT03070392 The first drug ever to extend life in metastatic uveal melanoma, and the first T-cell receptor-based medicine to win a phase 3. | Tebentafusp | Melanoma | Immunocore | 2021 | ||
JUPITER-02 NCT03581786 Brought immunotherapy to nasopharyngeal cancer, an Epstein-Barr-virus-driven cancer common in southern China, and won the first US approval for that disease. | Toripalimab | Head and neck squamous cell carcinoma | 2021 | |||
KEYNOTE-564 NCT03142334 The first drug to help kidney cancer patients live longer after surgery, and the first adjuvant immunotherapy in any solid tumour to show an overall survival gain. | Pembrolizumab | Renal cell carcinoma | Merck | 2021 | ||
KEYNOTE-716 NCT03553836 Extended adjuvant immunotherapy to thick node-negative melanomas, a group whose risk of relapse rivals stage III. | Pembrolizumab | Melanoma | Merck | 2021 | ||
KEYNOTE-775 / Study 309 NCT03517449 A pill that blocks tumour blood vessels plus immunotherapy extended survival after chemotherapy, including in tumours that immunotherapy alone does not touch. | Lenvatinib, Pembrolizumab | Endometrial cancer | Eisai, Merck | 2021 | ||
KEYNOTE-826 NCT03635567 Added nearly ten months of life for women with advanced cervical cancer and made immunotherapy part of first-line treatment. | Pembrolizumab, Bevacizumab, Cisplatin, Paclitaxel / nab-paclitaxel | Cervical cancer | Merck | 2021 | ||
LOTIS-2 NCT03589469 LOTIS-2 was the pivotal study for loncastuximab, a CD19 ADC with a DNA-crosslinking payload. | Loncastuximab tesirine | Diffuse large B-cell lymphoma | ADC Therapeutics | 2021 | ||
OlympiA NCT02032823 Showed that a year of a PARP inhibitor after standard treatment improves survival in women with inherited BRCA mutations. | Olaparib | Triple-negative breast cancer, HR-positive / HER2-negative breast cancer | AstraZeneca, , | 2021 | ||
PEACE-1 NCT01957436 PEACE-1 is the European triplet trial: abiraterone added to hormone therapy and docetaxel improves survival, especially in high-volume disease. | Abiraterone acetate, Docetaxel | Prostate cancer | UNICANCER, Gustave Roussy | 2021 | ||
POLARIX NCT03274492 The trial that improved on R-CHOP for the first time in twenty years, by swapping vincristine for an ADC. | Polatuzumab vedotin, Doxorubicin | Diffuse large B-cell lymphoma | Roche | 2021 | ||
TheraP (ANZUP 1603) NCT03392428 The randomised trial that first pitted a radioligand against chemotherapy and won on response, with fewer side effects. | Lutetium-177 vipivotide tetraxetan, Cabazitaxel | Prostate cancer | 2021 | |||
| Lutetium-177 vipivotide tetraxetan | Prostate cancer | Novartis | 2021 | |||
ZUMA-7 NCT03391466 The trial that moved CAR-T ahead of transplant as second-line treatment for early-relapsing large B-cell lymphoma, with a survival benefit. | Axicabtagene ciloleucel | Diffuse large B-cell lymphoma | Kite, Gilead | 2021 | ||
ADAURA NCT02511106 Showed a targeted pill after lung cancer surgery halves the risk of death, the first such result for a targeted therapy. | Osimertinib | Non-small-cell lung cancer | AstraZeneca | 2020 | ||
ASCENT NCT02574455 The trial that proved a TROP2 ADC could nearly double survival in heavily pretreated triple-negative breast cancer. | Sacituzumab govitecan | Triple-negative breast cancer | Gilead (Immunomedics) | 2020 | ||
CheckMate 649 NCT02872116 The trial that added immunotherapy to first-line stomach cancer chemotherapy; at five years, 16% of patients with PD-L1-rich tumours were alive versus 6%. | Nivolumab, CAPOX, FOLFOX | Gastric & gastro-oesophageal junction cancer, Oesophageal cancer | BMS | 2020 | ||
CheckMate 743 NCT02899299 CheckMate 743 was the first immunotherapy trial to lengthen survival in mesothelioma, and gave the first new first-line option in 16 years. | Nivolumab, Ipilimumab, Pemetrexed | Mesothelioma | BMS | 2020 | ||
CheckMate 9ER NCT03141177 Nivolumab with cabozantinib doubled progression-free survival versus sunitinib and lengthened life, with quality of life preserved. | Nivolumab, Cabozantinib, Sunitinib | Renal cell carcinoma | BMS, Exelixis | 2020 | ||
ClarIDHy NCT02989857 The first randomised trial of a targeted drug in bile duct cancer; it slowed the disease without shrinking it. | Ivosidenib | Biliary tract cancer | 2020 | |||
CROWN NCT03052608 The trial with the longest disease control ever seen for a targeted lung cancer pill: most patients still progression-free at five years. | Lorlatinib | Non-small-cell lung cancer | Pfizer | 2020 | ||
D-ALBA (GIMEMA LAL2116) NCT02744768 The trial that showed Ph-positive ALL can be treated with a pill plus an immunotherapy and no chemotherapy, with 95% of patients alive at 18 months. | Dasatinib, Blinatumomab | Acute lymphoblastic leukaemia | GIMEMA | 2020 | ||
DESKTOP III / ENGOT-ov20 NCT01166737 Operating again at first relapse, in carefully selected women, added about seven months of life. | — | Ovarian cancer | 2020 | |||
Euro Ewing 2012 NCT00987636 The American Ewing sarcoma regimen beat the European one in a head-to-head trial, unifying practice. | Doxorubicin, Ifosfamide | Sarcomas | 2020 | |||
FIGHT-202 NCT02924376 FIGHT-202 is the single-arm study that produced the first targeted approval in bile duct cancer. | Pemigatinib | Biliary tract cancer | Incyte | 2020 | ||
IMbrave150 NCT03434379 The trial that ended the sorafenib era: immunotherapy plus an anti-VEGF antibody became the new first-line standard for liver cancer. | Atezolizumab | Hepatocellular carcinoma | Roche | 2020 | ||
INVICTUS NCT03353753 Ripretinib kept fourth-line GIST under control six times longer than placebo and improved survival. | Ripretinib | Sarcomas | 2020 | |||
JAVELIN Bladder 100 NCT02603432 Starting immunotherapy right after chemotherapy, rather than waiting for relapse, lengthened survival by about seven months. | Avelumab | Bladder & urothelial cancer | , Pfizer | 2020 | ||
KEYNOTE-177 NCT02563002 The trial that made immunotherapy alone, with no chemotherapy, the first treatment for the 5% of bowel cancers with a broken DNA spell-checker. Over half of patients were alive at five years. | Pembrolizumab | Colorectal cancer | Merck | 2020 | ||
KEYNOTE-204 NCT02684292 In KEYNOTE-204, PD-1 blockade beat the CD30 ADC head to head in relapsed Hodgkin lymphoma. | Pembrolizumab, Brentuximab vedotin | Hodgkin lymphoma | Merck | 2020 | ||
KEYNOTE-355 NCT02819518 Established immunotherapy plus chemotherapy as first-line treatment for metastatic triple-negative breast cancer with PD-L1 expression. | Pembrolizumab | Triple-negative breast cancer | Merck | 2020 | ||
KEYNOTE-522 NCT03036488 The trial that added immunotherapy to pre-surgery chemotherapy for triple-negative breast cancer and, uniquely, improved survival. | Pembrolizumab, Carboplatin, Paclitaxel / nab-paclitaxel | Triple-negative breast cancer | Merck | 2020 | ||
KEYNOTE-590 NCT03189719 Added immunotherapy to first-line chemotherapy for all types of oesophageal cancer, with a survival benefit that held at five years. | Pembrolizumab | Oesophageal cancer | Merck | 2020 | ||
L-MIND NCT02399085 The small single-arm study that got tafasitamab approved for people too frail for transplant. | Tafasitamab, Lenalidomide | Diffuse large B-cell lymphoma | MorphoSys | 2020 | ||
monarchE NCT03155997 The first CDK4/6 inhibitor to reduce recurrence after surgery in high-risk hormone-positive breast cancer. | Abemaciclib | HR-positive / HER2-negative breast cancer | Eli Lilly | 2020 | ||
Naxitamab Study 201 NCT03363373 Study 201 was the pivotal single-arm study behind naxitamab's approval. | Naxitamab | Neuroblastoma | 2020 | |||
PROfound NCT02987543 The first biomarker-selected trial in prostate cancer to improve survival, using a PARP inhibitor in men with BRCA-type mutations. | Olaparib | Prostate cancer | AstraZeneca, Merck | 2020 | ||
proPSMA ANZCTR12617000005358 Proved PSMA PET is far more accurate than conventional scans for staging, with less radiation. | — | Prostate cancer | Peter MacCallum Cancer Centre, | 2020 | ||
RxPONDER (SWOG S1007) NCT01272037 Postmenopausal women with a few positive lymph nodes and a low gene-test score can skip chemotherapy; premenopausal women still benefit from it. | Oncotype DX | HR-positive / HER2-negative breast cancer | SWOG, NCI | 2020 | ||
SANET-ep and SANET-p NCT02588170 In the SANET trials, a Chinese anti-angiogenic pill slowed both gut and pancreatic neuroendocrine tumours, but its US application was refused. | — | Neuroendocrine tumours | 2020 | |||
VIALE-A NCT02993523 The trial that gave older AML patients a real treatment: adding venetoclax to azacitidine doubled remission rates and extended survival. | Venetoclax, Azacitidine | Acute myeloid leukaemia | AbbVie, Genentech | 2020 | ||
ADMIRAL NCT02421939 Showed a pill beats intensive salvage chemotherapy in relapsed FLT3-mutated AML, with fewer days in hospital. | Gilteritinib | Acute myeloid leukaemia | Astellas | 2019 | ||
ARCHES NCT02677896 Brought enzalutamide into first-line metastatic treatment, with an overall survival benefit confirmed in 2021. | Enzalutamide | Prostate cancer | Astellas, Pfizer | 2019 | ||
CASPIAN NCT03043872 Confirmed that adding a PD-L1 blocker to first-line chemotherapy helps in small-cell lung cancer, and showed that adding a second immunotherapy did not help further. | Durvalumab, Tremelimumab, Platinum + etoposide | Small-cell lung cancer | AstraZeneca | 2019 | ||
CLL14 NCT02242942 One year of two targeted drugs, then nothing: more than half of patients were still progression-free six years later, off all treatment. | Venetoclax, Obinutuzumab | Chronic lymphocytic leukaemia | , Roche, AbbVie | 2019 | ||
ELEVATE-TN NCT02475097 Six years on, most patients on acalabrutinib have still not progressed, and adding obinutuzumab also improved survival. | Acalabrutinib, Obinutuzumab | Chronic lymphocytic leukaemia | AstraZeneca, | 2019 | ||
HER2CLIMB NCT02614794 The first trial to prove a drug helps HER2-positive brain metastases, extending survival by four and a half months overall. | Tucatinib, Trastuzumab | HER2-positive breast cancer | Seagen (Pfizer) | 2019 | ||
KEYNOTE-048 NCT02358031 Made immunotherapy the first treatment for advanced head and neck cancer, alone for PD-L1-rich tumours and with chemotherapy for the rest. | Pembrolizumab | Head and neck squamous cell carcinoma | Merck | 2019 | ||
KEYNOTE-426 NCT02853331 The first immunotherapy plus targeted-pill combination for kidney cancer, improving survival across all risk groups. | Pembrolizumab, Axitinib, Sunitinib | Renal cell carcinoma | Merck | 2019 | ||
PAOLA-1 / ENGOT-ov25 NCT02477644 Adding olaparib to bevacizumab maintenance roughly doubled progression-free time in tumours with faulty DNA repair, and improved survival in that group. | Olaparib, Bevacizumab | Ovarian cancer | ARCAGY-GINECO, AstraZeneca | 2019 | ||
PERSEPHONE NCT00712140 Six months of trastuzumab was almost as good as twelve, with half the heart toxicity, though twelve remains the global standard. | Trastuzumab | HER2-positive breast cancer | Cancer Research UK | 2019 | ||
STELLAR NCT02397928 STELLAR is the small single-arm study behind the device approval of tumour treating fields in mesothelioma. | Optune / Optune Pax, Pemetrexed | Mesothelioma | Novocure | 2019 | ||
CELESTIAL NCT01908426 Cabozantinib extended survival in previously treated liver cancer, including after two prior therapies. | Cabozantinib | Hepatocellular carcinoma | Exelixis | 2018 | ||
CheckMate 214 NCT02231749 CheckMate 214 is the trial that brought dual immunotherapy to kidney cancer, with a survival advantage still visible eight years later and a fifth of patients in long-term remission. | Nivolumab, Ipilimumab, Sunitinib | Renal cell carcinoma | BMS | 2018 | ||
COLUMBUS NCT01909453 COLUMBUS tested encorafenib-binimetinib, the third BRAF/MEK doublet, which has the longest median survival of the class and less fever than dabrafenib-trametinib. | Encorafenib, Binimetinib, Vemurafenib | Melanoma | , Pfizer | 2018 | ||
CPX-351 Study 301 NCT01696084 Repackaging two old chemotherapies into liposomes doubled five-year survival in older adults with the worst kinds of AML. | CPX-351, Cytarabine + anthracycline | Acute myeloid leukaemia | , Jazz | 2018 | ||
ELIANA NCT02435849 ELIANA was the global trial behind the first CAR-T approval: four in five children with no options left went into remission, and many stayed there for years. | Tisagenlecleucel | Acute lymphoblastic leukaemia | Novartis | 2018 | ||
IMpower133 NCT02763579 IMpower133 was the first trial in decades to lengthen survival in extensive-stage small-cell lung cancer, by adding immunotherapy to chemotherapy. | Atezolizumab, Platinum + etoposide | Small-cell lung cancer | Roche | 2018 | ||
KATHERINE NCT01772472 Switching to an ADC after surgery when pre-surgery treatment left cancer behind halved the risk of recurrence and improved survival. | Trastuzumab emtansine, Trastuzumab | HER2-positive breast cancer | Roche, Genentech | 2018 | ||
OVHIPEC-1 NCT00426257 Washing the abdomen with heated chemotherapy during interval surgery extended survival by about a year. | — | Ovarian cancer | Netherlands Cancer Institute | 2018 | ||
PORTEC-3 NCT00411138 Adding chemotherapy to radiation after surgery helped women with high-risk endometrial cancer, especially those whose tumours have a broken p53 gene. | — | Endometrial cancer | 2018 | |||
PRECISION NCT02380027 PRECISION proved that an MRI first finds more dangerous cancers with fewer biopsies. | — | Prostate cancer | 2018 | |||
PRODIGE 24 / CCTG PA6 NCT01526135 Showed that giving the strong four-drug chemotherapy after pancreatic surgery adds years of life for fit patients. | FOLFIRINOX / mFOLFIRINOX | Pancreatic ductal adenocarcinoma | UNICANCER, | 2018 | ||
REFLECT NCT01761266 Lenvatinib matched sorafenib on survival with more tumour shrinkage, giving a second first-line option after a decade. | Lenvatinib, Sorafenib | Hepatocellular carcinoma | Eisai | 2018 | ||
ROAR (anaplastic thyroid cancer cohort) NCT02034110 Turned the most lethal thyroid cancer from a months-long death sentence into a treatable disease for the third of patients whose tumours carry a BRAF mutation. | Dabrafenib + trametinib | Thyroid cancer | Novartis | 2018 | ||
SOLAR-1 NCT02437318 The first trial to show a PI3K inhibitor helps in breast cancers carrying a PIK3CA mutation, at the cost of high blood sugar and rash. | Alpelisib | HR-positive / HER2-negative breast cancer | Novartis | 2018 | ||
SOLO-1 NCT01844986 Two years of an olaparib pill after chemotherapy made a large share of women with BRCA-mutated ovarian cancer long-term survivors. | Olaparib | Ovarian cancer | AstraZeneca, Merck | 2018 | ||
TAILORx NCT00310180 Showed that most women with the commonest breast cancer can safely skip chemotherapy if a gene test says their risk is low or intermediate. | Oncotype DX | HR-positive / HER2-negative breast cancer | ECOG-ACRIN, NCI | 2018 | ||
APHINITY NCT01358877 Adding pertuzumab after surgery helps women whose cancer had reached the lymph nodes, by a few percentage points, and does not help those with node-negative disease. | Pertuzumab, Trastuzumab | HER2-positive breast cancer | Roche, | 2017 | ||
COMBI-AD NCT01682083 A year of two targeted pills after surgery halves the risk of relapse in BRAF-mutant melanoma, and the benefit is still visible a decade later. | Dabrafenib + trametinib | Melanoma | Novartis | 2017 | ||
ECHELON-1 NCT01712490 Swapping bleomycin for the CD30 ADC in first-line chemotherapy improved survival in advanced Hodgkin lymphoma, the first frontline survival gain in decades. | Brentuximab vedotin, Doxorubicin | Hodgkin lymphoma | Seagen, Takeda | 2017 | ||
FIRES & SENTOR (sentinel node mapping) NCT01673022 Mapping and removing only the first draining lymph nodes finds spread as reliably as removing all of them, with far less lymphoedema. | — | Endometrial cancer | 2017 | |||
ISG-STS 1001 NCT01710176 Standard anthracycline-ifosfamide before surgery beat subtype-tailored chemotherapy, and indirectly showed neoadjuvant chemotherapy helps high-risk sarcoma. | Ifosfamide | Sarcomas | 2017 | |||
LATITUDE NCT01715285 LATITUDE established abiraterone at first metastatic diagnosis for high-risk disease. | Abiraterone acetate | Prostate cancer | Janssen | 2017 | ||
MONARCH 3 NCT02246621 Abemaciclib roughly doubled the time to progression when added to an aromatase inhibitor; the survival gain of about 13 months narrowly missed statistical significance. | Abemaciclib | HR-positive / HER2-negative breast cancer | Eli Lilly | 2017 | ||
MSLT-II NCT00297895 Showed that removing all the remaining lymph nodes after a positive sentinel node does not help patients live longer, ending a routine operation. | — | Melanoma | , NCI | 2017 | ||
PACIFIC NCT02125461 Made a year of immunotherapy after chemoradiation the standard for stage III lung cancer, with a survival benefit that held at five years. | Durvalumab | Non-small-cell lung cancer | AstraZeneca | 2017 | ||
RATIFY (CALGB 10603) NCT00651261 The trial that made FLT3 the first targetable mutation in AML: adding midostaurin to chemotherapy tripled median survival. | Midostaurin, Cytarabine + anthracycline | Acute myeloid leukaemia | Alliance, Novartis | 2017 | ||
| Regorafenib | Hepatocellular carcinoma | Bayer | 2017 | |||
TOWER NCT02013167 The first randomised proof that a T-cell engager beats chemotherapy: blinatumomab nearly doubled survival in relapsed adult ALL. | Blinatumomab | Acute lymphoblastic leukaemia | Amgen | 2017 | ||
CheckMate 141 NCT02105636 The first immunotherapy to extend survival in head and neck cancer, in patients who had progressed on platinum within six months. | Nivolumab | Head and neck squamous cell carcinoma | BMS | 2016 | ||
CheckMate 205 NCT02181738 Established PD-1 blockade in relapsed Hodgkin lymphoma with ~70% response rates and seeded the frontline nivolumab-AVD idea. | Nivolumab | Hodgkin lymphoma | BMS | 2016 | ||
COG ANBL0532 NCT00567567 COG ANBL0532 showed that two transplants in a row beat one in high-risk neuroblastoma. | — | Neuroblastoma | Children's Oncology Group | 2016 | ||
INO-VATE ALL NCT01564784 An antibody-drug conjugate put four in five relapsed ALL patients into remission versus fewer than one in three with chemotherapy. | Inotuzumab ozogamicin | Acute lymphoblastic leukaemia | Pfizer | 2016 | ||
KEYNOTE-024 & KEYNOTE-189 NCT02142738 The two trials that made immunotherapy, alone or with chemotherapy, the first treatment for most advanced lung cancers. | Pembrolizumab | Non-small-cell lung cancer | Merck | 2016 | ||
MAPS NCT00651456 In MAPS, adding the anti-VEGF antibody bevacizumab to chemotherapy lengthened survival by about three months, the first improvement after pemetrexed. | Pemetrexed | Mesothelioma | 2016 | |||
MONALEESA-2 NCT01958021 The first CDK4/6 inhibitor trial to show that adding the pill to hormone therapy makes women live longer, by about a year. | Ribociclib | HR-positive / HER2-negative breast cancer | Novartis | 2016 | ||
RATHL NCT00678327 Showed that patients whose PET scan is clear after two cycles can safely drop bleomycin and its lung toxicity. | — | Hodgkin lymphoma | , Cancer Research UK | 2016 | ||
STAMPEDE NCT00268476 STAMPEDE is the longest-running platform trial in oncology, and showed that both docetaxel and abiraterone extend life when started at first diagnosis of metastatic disease. | Abiraterone acetate, Docetaxel | Prostate cancer | 2016 | |||
AETHERA NCT01100502 In AETHERA, a year of the CD30 ADC after transplant halved relapse risk in high-risk patients. | Brentuximab vedotin | Hodgkin lymphoma | Seagen | 2015 | ||
APT (adjuvant paclitaxel-trastuzumab) NCT00542451 For small HER2-positive tumours, a gentle regimen of weekly paclitaxel plus trastuzumab gives excellent cure rates, avoiding harsher chemotherapy. | Paclitaxel / nab-paclitaxel, Trastuzumab | HER2-positive breast cancer | 2015 | |||
CHAARTED (E3805) NCT00309985 The first trial to show chemotherapy at the start of hormone therapy prolongs life in metastatic prostate cancer. | Docetaxel | Prostate cancer | ECOG-ACRIN | 2015 | ||
CheckMate 067 NCT01844505 The trial with the longest immunotherapy follow-up: about half of patients on the combination are alive at 10 years. | Nivolumab, Ipilimumab | Melanoma | BMS | 2015 | ||
EF-14 NCT00916409 The trial that made a wearable electric-field device part of glioblastoma care, extending median survival by about five months. | Optune / Optune Pax, Temozolomide | Glioma & glioblastoma | Novocure | 2015 | ||
KEYNOTE-006 NCT01866319 The trial that made PD-1 blockade the first choice over the older CTLA-4 drug, with a third of patients alive at ten years. | Pembrolizumab, Ipilimumab | Melanoma | Merck | 2015 | ||
SELECT NCT01321554 Turned lenvatinib into the main drug for thyroid cancers that no longer take up radioactive iodine, quadrupling the time before the disease grew. | — | Thyroid cancer | Eisai | 2015 | ||
CLARINET NCT00353496 Lanreotide more than halved the risk of progression in gut and pancreatic neuroendocrine tumours. | Somatostatin analogues | Neuroendocrine tumours | Ipsen | 2014 | ||
RAINBOW NCT01170663 Made ramucirumab plus paclitaxel the standard second-line stomach cancer treatment, a role it held for a decade until Enhertu and Claudin drugs arrived. | Ramucirumab, Paclitaxel / nab-paclitaxel | Gastric & gastro-oesophageal junction cancer | Eli Lilly | 2014 | ||
RESONATE NCT01578707 The trial that made a daily pill the standard for relapsed CLL, cutting the risk of progression by nearly 80%. | Ibrutinib | Chronic lymphocytic leukaemia | , Janssen | 2014 | ||
SOFT & TEXT NCT00066690 For younger women, shutting down the ovaries and adding an aromatase inhibitor prevents more recurrences than tamoxifen alone, with the largest gains in the highest-risk women. | Exemestane, Tamoxifen, Goserelin / leuprolide | HR-positive / HER2-negative breast cancer | 2014 | |||
| Radium-223 dichloride | Prostate cancer | Bayer, | 2013 | |||
DECISION NCT00984282 The first drug approved for thyroid cancers that stopped responding to radioactive iodine. | — | Thyroid cancer | Bayer | 2013 | ||
ALFA-0701 NCT00927498 ALFA-0701 was the trial that rescued the first ADC: giving gemtuzumab in three small doses with chemotherapy improved event-free survival without the toxicity that got it withdrawn. | Gemtuzumab ozogamicin, Cytarabine + anthracycline | Acute myeloid leukaemia | 2012 | |||
CROSS NTR487 The trial that made chemotherapy plus radiation before surgery the standard for oesophageal cancer; the survival gain was still there ten years later. | Carboplatin, Paclitaxel / nab-paclitaxel | Oesophageal cancer | 2012 | |||
HiLo NCT00415233 Showed a third of the usual radioactive iodine dose ablates the thyroid remnant just as well, with fewer side effects and less time in isolation. | — | Thyroid cancer | Cancer Research UK | 2012 | ||
SSGXVIII/AIO (adjuvant imatinib in GIST) NCT00116935 In SSGXVIII/AIO, three years of imatinib after surgery, rather than one, improved survival in high-risk GIST. | Imatinib | Sarcomas | 2012 | |||
CLEOPATRA NCT00567190 Adding a second HER2 antibody, pertuzumab, extended survival by 16 months, the largest gain ever seen in metastatic breast cancer at the time. | Pertuzumab, Trastuzumab, Docetaxel | HER2-positive breast cancer | Roche, Genentech | 2011 | ||
NLST & NELSON (low-dose CT screening) The two trials that proved a yearly low-dose CT scan cuts lung cancer deaths in heavy smokers. | — | Non-small-cell lung cancer | 2011 | |||
RADIANT-3 and RADIANT-4 NCT00510068 The two trials that made everolimus a standard pill for pancreatic, lung and gut neuroendocrine tumours. | Everolimus | Neuroendocrine tumours | Novartis | 2011 | ||
ABC-02 The 2010 UK trial that gave bile duct cancer its first standard chemotherapy. | Gemcitabine + cisplatin | Biliary tract cancer | Cancer Research UK | 2010 | ||
COG ANBL0032 NCT00026312 The trial that made anti-GD2 immunotherapy standard for children with high-risk neuroblastoma, improving survival by about 20 points. | Dinutuximab (ch14.18) / dinutuximab beta | Neuroblastoma | Children's Oncology Group | 2010 | ||
ToGA NCT01041404 The trial that brought the breast-cancer drug Herceptin to stomach cancer, the first targeted therapy to improve survival in this disease. | Trastuzumab | Gastric & gastro-oesophageal junction cancer | Roche | 2010 | ||
CRYSTAL & FIRE-3 The trials that established EGFR antibodies in bowel cancer and discovered they only work when the RAS gene is normal. | Cetuximab, Bevacizumab, FOLFIRI | Colorectal cancer | 2009 | |||
PROMID NCT00171873 Showed for the first time that a hormone-suppressing injection also slows neuroendocrine tumour growth. | Somatostatin analogues | Neuroendocrine tumours | Novartis, | 2009 | ||
EXTREME NCT00122460 The regimen that defined first-line treatment for advanced head and neck cancer from 2008 until immunotherapy replaced it. | — | Head and neck squamous cell carcinoma | 2008 | |||
| Sorafenib | Hepatocellular carcinoma | Bayer | 2008 | |||
EORTC 26981 / NCIC CE.3 (Stupp trial) NCT00006353 The 2005 trial that set the treatment every glioblastoma patient still receives. Nothing has replaced it in twenty years. | Temozolomide | Glioma & glioblastoma | EORTC, | 2005 | ||
HERA, NSABP B-31 & NCCTG N9831 (adjuvant trastuzumab) NCT00045032 Three trials reported together in 2005 showed that a year of trastuzumab after surgery cuts the risk of dying from HER2-positive breast cancer by about a third. | Trastuzumab | HER2-positive breast cancer | 2005 | |||
INT-0091 (Ewing sarcoma) NCT00002516 Adding ifosfamide and etoposide improved cure rates for localised Ewing sarcoma, setting the backbone still used today. | Doxorubicin, Ifosfamide | Sarcomas | 2003 | |||
PATHFINDER 2 NCT05155605 PATHFINDER 2 is the US real-world study of the Galleri test that forms the core of its FDA submission. | Galleri | — | GRAIL | 2025 | ||
MIMic-01: healthy-donor FMT plus anti-PD-1, first-line melanoma NCT03772899 Twenty melanoma patients took stool capsules from healthy donors before starting immunotherapy. Thirteen responded, more than usually expected, and the transplant was safe. | Nivolumab, Pembrolizumab | Melanoma | 2023 | |||
FMT plus pembrolizumab in anti-PD-1-refractory melanoma (Pittsburgh) NCT03341143 Fifteen melanoma patients whose immunotherapy had failed received a stool transplant from someone whose immunotherapy had worked, then restarted the drug. Six of them benefited, including three with lasting responses. | Pembrolizumab | Melanoma | UPMC Hillman Cancer Center, NCI | 2021 | ||
ProtecT NCT02044172 Showed that men with screen-detected prostate cancer die of it rarely, whether monitored or treated, establishing active surveillance. | — | Prostate cancer | 2016 | |||
RTOG 0129 (HPV analysis) NCT00047008 The analysis that showed HPV-positive throat cancers are a different, far more curable disease, launching two decades of de-escalation research. | — | Head and neck squamous cell carcinoma | , NCI | 2010 | ||
NHS-Galleri ISRCTN91431511 The largest cancer screening trial ever run, testing whether a multi-cancer blood test reduces late-stage diagnoses. | Galleri | — | , GRAIL | — | ||
MOUNTAINEER & MOUNTAINEER-03 NCT03043313 A HER2 pill plus antibody gave durable responses in the 3-5% of bowel cancers driven by HER2, and is now being tested as first-line treatment. | Tucatinib, Trastuzumab, FOLFOX | Colorectal cancer | Pfizer (Seagen) | 2022 | ||
ACTION-1 NCT05477576 Tests whether an actinium alpha-radioligand rescues neuroendocrine tumours after lutetium therapy fails. | Actinium-225 DOTATATE, Lutetium-177 dotatate | Neuroendocrine tumours | BMS (RayzeBio) | — | ||
AHOD2131 (COG / NCTN) NCT05675410 Asks whether early-stage Hodgkin lymphoma in children and adults can be treated with immunotherapy instead of some chemotherapy and radiation. | Brentuximab vedotin, Nivolumab | Hodgkin lymphoma | Children's Oncology Group, NCI | — | ||
AlphaBreak (FPI-2265) & AcTION (225Ac-PSMA-617) NCT06402331 The phase 3 trials that will decide whether alpha-emitting PSMA drugs become the next step after Pluvicto. | Actinium-225 PSMA agents | Prostate cancer | — | |||
BELLWAVE-011 NCT06136559 BELLWAVE-011 is Merck's bid to enter frontline CLL with a reversible BTK inhibitor, head to head against two approved covalent drugs. | Nemtabrutinib, Ibrutinib, Acalabrutinib | Chronic lymphocytic leukaemia | Merck | — | ||
CaDAnCe-304 CaDAnCe-304 is the first phase 3 of a protein degrader in leukaemia, pitting BTK destruction against the best available BTK inhibitor. | BGB-16673, Pirtobrutinib | Chronic lymphocytic leukaemia | BeOne (BeiGene) | — | ||
CAMBRIA-1 & CAMBRIA-2 NCT05952557 Two very large trials testing whether an oral SERD should replace today's adjuvant hormone pills, either from the start or as a switch. | Camizestrant | HR-positive / HER2-negative breast cancer | AstraZeneca | — | ||
CARTITUDE-5 NCT04923893 Tests CAR-T as frontline consolidation instead of ongoing lenalidomide in patients not having a transplant. | Ciltacabtagene autoleucel | Multiple myeloma | Janssen | — | ||
DeLLphi-305 NCT06211036 Tests whether adding the DLL3 T-cell engager to first-line maintenance can push median survival in extensive-stage disease past two years, as the phase 1b hinted. | Tarlatamab, Durvalumab | Small-cell lung cancer | Amgen | — | ||
FIRST-308 The first phase 3 trial for patients whose bile duct cancer has outgrown the existing FGFR drugs. | Tinengotinib | Biliary tract cancer | — | |||
FORTIFI-HN01 NCT06788990 A bifunctional antibody that blocks EGFR and soaks up TGF-beta, aiming to make HPV-negative head and neck tumours respond to immunotherapy. | Ficerafusp alfa, Pembrolizumab | Head and neck squamous cell carcinoma | — | |||
GOLSEEK-1 NCT06356129 Tests whether a potent oral degrader added to R-CHOP raises cure rates in high-risk disease. | Golcadomide | Diffuse large B-cell lymphoma | BMS | — | ||
HERIZON-BTC-302 NCT06282575 Tests whether the HER2 bispecific antibody should be given from the start in HER2-positive bile duct cancer. | Zanidatamab, Gemcitabine + cisplatin | Biliary tract cancer | Jazz, Zymeworks | — | ||
IDeate-Lung02 NCT06203210 IDeate-Lung02 is the pivotal trial for the B7-H3 ADC that produced some of the highest response rates ever seen in relapsed small-cell lung cancer. | Ifinatamab deruxtecan, Topotecan, Lurbinectedin | Small-cell lung cancer | Daiichi Sankyo, Merck | — | ||
IZABRIGHT-Breast01 NCT06926868 IZABRIGHT-Breast01 is the global first-line trial of the EGFR×HER3 bispecific ADC in triple-negative breast cancer. | Izalontamab brengitecan | Triple-negative breast cancer | BMS | — | ||
LiGeR-HN1 NCT06525220 Tests whether a two-armed antibody against EGFR and a stem-cell marker can lift first-line immunotherapy results in head and neck cancer. | Petosemtamab, Pembrolizumab | Head and neck squamous cell carcinoma | Merus (Genmab) | — | ||
MEVPRO-1 NCT06551324 MEVPRO-1 is the phase 3 trial of the first EZH2 inhibitor combination in prostate cancer. | Mevrometostat, Enzalutamide | Prostate cancer | Pfizer | — | ||
myeloMATCH NCT05564390 myeloMATCH is a US-wide master trial that sequences every new leukaemia within days and routes the patient to the right experimental arm, then re-routes them as MRD results come in. | Ziftomenib, CPX-351, Venetoclax | Acute myeloid leukaemia | NCI, | — | ||
OptimICE-pCR (A012103) NCT05812807 Asks whether patients whose cancer completely disappeared before surgery still need a year of immunotherapy afterwards. | Pembrolizumab | Triple-negative breast cancer | Alliance, NCI | — | ||
PRISM-MEL-301 NCT06112314 Tests whether a PRAME-directed T-cell engager adds to first-line immunotherapy in ordinary skin melanoma, following tebentafusp's success in the eye form. | Brenetafusp, Nivolumab | Melanoma | Immunocore | — | ||
REJOICE-Ovarian01 NCT06161025 Tests whether a CDH6-targeting ADC with Enhertu's payload can beat chemotherapy in resistant ovarian cancer. | Raludotatug deruxtecan | Ovarian cancer | Daiichi Sankyo, Merck | — | ||
SCARLET (SWOG S2212) NCT05929768 Tests whether the anthracycline can be dropped from TNBC pre-surgery treatment without losing effect. | Pembrolizumab, Carboplatin | Triple-negative breast cancer | SWOG, NCI | — | ||
SENTICOL III NCT03386734 Tests whether removing only the first draining lymph nodes is safe enough to spare women the lymphoedema of full node removal. | — | Cervical cancer | ARCAGY-GINECO, | — | ||
TeliMET NSCLC-01 NCT04928846 The confirmatory trial for the first c-MET ADC, which was approved early on response rate alone. | Telisotuzumab vedotin | Non-small-cell lung cancer | AbbVie | — | ||
TROPION-Breast05 NCT06103864 Tests whether Dato-DXd plus a PD-L1 blocker beats today's immunotherapy-chemotherapy standard. | Datopotamab deruxtecan, Durvalumab, Pembrolizumab | Triple-negative breast cancer | AstraZeneca, Daiichi Sankyo | — | ||
waveLINE-003 NCT05139017 waveLINE-003 tests a ROR1-directed ADC added to standard salvage chemotherapy. | Zilovertamab vedotin | Diffuse large B-cell lymphoma | Merck | — | ||
RAINFOL-01 (Rina-S) NCT05579366 A next-generation folate-receptor ADC that works even in tumours with low folate receptor, with responses in both ovarian and endometrial cancer. | Rinatabart sesutecan | Ovarian cancer, Endometrial cancer | Genmab | 2025 | ||
CIRCULATE-Japan (GALAXY / VEGA / ALTAIR) Japan's national programme tests whether a blood test after surgery should decide who gets chemotherapy, and whether treating a positive test early helps. | Signatera, Trifluridine/tipiracil, CAPOX | Colorectal cancer | 2023 | |||
Add-Aspirin NCT02804815 Add-Aspirin is an 11,000-patient trial across four common cancers in the UK, Ireland and India asking whether cheap daily aspirin after curative treatment stops cancer coming back. | — | Colorectal cancer, HR-positive / HER2-negative breast cancer, Gastric & gastro-oesophageal junction cancer, Oesophageal cancer, Prostate cancer | , Cancer Research UK, , | — | ||
ASCENT-05 / OptimICE-RD (AFT-65, GBG 119, NSABP B-63) NCT05633654 Tests whether giving a TROP2 ADC after surgery can cure more of the triple-negative patients whose cancer survived pre-surgery chemo-immunotherapy. | Sacituzumab govitecan, Pembrolizumab | Triple-negative breast cancer | Gilead, | — | ||
CELESTIAL-TNCLL NCT06073821 CELESTIAL-TNCLL tests the next-generation all-oral doublet against the current fixed-duration standard, with results expected from late 2026. | Sonrotoclax, Zanubrutinib, Venetoclax, Obinutuzumab | Chronic lymphocytic leukaemia | BeOne (BeiGene) | — | ||
COG ANBL1531 NCT03126916 COG ANBL1531 is the current North American high-risk trial, adding targeted radiation during induction and an ALK pill for children whose tumours carry ALK mutations. | 131I-MIBG (iobenguane I-131) therapy, Lorlatinib | Neuroblastoma | Children's Oncology Group | — | ||
EPCORE DLBCL-2 NCT05578976 Tests whether adding a bispecific to first-line chemotherapy cures more patients up front. | Epcoritamab | Diffuse large B-cell lymphoma | Genmab, AbbVie | — | ||
iStopMM NCT03327597 iStopMM screened an entire nation for the precursor of myeloma, to learn whether finding it early saves lives or just causes worry. | — | Multiple myeloma | — | |||
TROPION-Breast03 NCT05629585 TROPION-Breast03 is the Dato-DXd counterpart to ASCENT-05: an ADC, with or without immunotherapy, for triple-negative patients with leftover cancer at surgery. | Datopotamab deruxtecan, Durvalumab, Pembrolizumab | Triple-negative breast cancer | AstraZeneca, Daiichi Sankyo | — | ||
| Xaluritamig, Cabazitaxel | Prostate cancer | Amgen | — | |||
EPCORE DLBCL-1 NCT04628494 Epcoritamab delayed progression but did not clearly extend life versus chemotherapy in relapsed lymphoma, missing its US primary endpoint in January 2026. | Epcoritamab | Diffuse large B-cell lymphoma | Genmab, AbbVie | 2026 | ||
HARMONi-3 NCT05899608 The global test of whether ivonescimab plus chemotherapy beats the Keytruda-chemotherapy standard. An interim look in May 2026 fell short on progression; final results are due later in 2026. | Ivonescimab, Pembrolizumab | Non-small-cell lung cancer | Summit Therapeutics | 2026 | ||
BWEL (Breast Cancer Weight Loss, Alliance A011401) NCT02750826 BWEL is a trial of more than 3,000 women testing whether losing weight after breast cancer treatment reduces recurrence. The programme achieved weight loss; the effect on recurrence was not clearly shown at the first analysis. | — | HR-positive / HER2-negative breast cancer, HER2-positive breast cancer | Alliance for Clinical Trials in Oncology, NCI | 2025 | ||
FORTITUDE-101 NCT05052801 A new target, FGFR2b, showed a survival gain at first look that shrank with longer follow-up, and the trial did not include the immunotherapy patients now routinely get. | Bemarituzumab, FOLFOX | Gastric & gastro-oesophageal junction cancer | Amgen | 2025 | ||
HERTHENA-Lung02 NCT05338970 A HER3 ADC delayed progression by only a few days more than chemotherapy and did not extend survival, so its US application was withdrawn. | Patritumab deruxtecan | Non-small-cell lung cancer | Daiichi Sankyo, Merck | 2025 | ||
NRG-HN002 & NRG-HN005 (HPV+ de-escalation) NCT03952585 Attempts to give HPV-positive throat cancer patients less radiation fell short: the standard dose remained better, so de-escalation is not yet routine. | — | Head and neck squamous cell carcinoma | NRG Oncology, NCI | 2025 | ||
VERITAC-2 NCT05654623 The trial that got the first PROTAC approved, with benefit only in tumours with ESR1 mutations. | Vepdegestrant | HR-positive / HER2-negative breast cancer | Arvinas, Pfizer | 2025 | ||
BEAT-meso (ETOP 13-18) NCT03762018 Adding atezolizumab to bevacizumab-chemotherapy slowed progression but did not lengthen survival overall; it did help patients with non-epithelioid tumours. | Atezolizumab, Pemetrexed | Mesothelioma | 2024 | |||
EMERALD-1 NCT03778957 The first trial to show that adding immunotherapy and an anti-VEGF drug to TACE delays progression in intermediate-stage liver cancer. | Durvalumab | Hepatocellular carcinoma | AstraZeneca | 2024 | ||
LEAP-012 NCT04246177 Adding lenvatinib and pembrolizumab to TACE delayed progression by four and a half months but did not help patients live longer. | Lenvatinib, Pembrolizumab | Hepatocellular carcinoma | Merck, Eisai | 2024 | ||
SPLASH NCT04647526 In SPLASH, a second PSMA radioligand improved progression-free survival but not, so far, survival. | 177Lu-PSMA-I&T | Prostate cancer | Lantheus (POINT Biopharma) | 2024 | ||
STARGLO NCT04408638 A bispecific plus chemotherapy improved survival in relapsed lymphoma, but the FDA rejected it because most patients were enrolled in Asia and the US-relevant subgroup did not clearly benefit. | Glofitamab | Diffuse large B-cell lymphoma | Roche | 2024 | ||
DUO-O / ENGOT-ov46 NCT03737643 Adding immunotherapy and olaparib to bevacizumab slowed progression but has not improved survival, and the regimen is not approved. | Durvalumab, Olaparib, Bevacizumab | Ovarian cancer | AstraZeneca | 2023 | ||
HER2CLIMB-02 NCT03975647 Adding tucatinib to Kadcyla helped a little, mostly in patients with brain metastases, but did not extend survival. | Tucatinib, Trastuzumab emtansine | HER2-positive breast cancer | Seagen (Pfizer) | 2023 | ||
TROPION-Breast01 NCT05104866 The trial that got Datroway approved in hormone-positive breast cancer, though it did not extend overall survival. | Datopotamab deruxtecan | HR-positive / HER2-negative breast cancer | AstraZeneca, Daiichi Sankyo | 2023 | ||
TROPION-Lung01 NCT04656652 A TROP2 ADC helped in non-squamous lung cancer but not squamous, and the overall survival result fell short. | Datopotamab deruxtecan | Non-small-cell lung cancer | AstraZeneca, Daiichi Sankyo | 2023 | ||
COSMIC-313 NCT03937219 The first triplet trial in kidney cancer slowed progression but did not help patients live longer, and added toxicity. | Cabozantinib, Nivolumab, Ipilimumab | Renal cell carcinoma | Exelixis | 2022 | ||
MAGNITUDE NCT03748641 PARP inhibitor plus abiraterone helped men with BRCA mutations and did nothing for those without, settling a debate. | Niraparib, Abiraterone acetate | Prostate cancer | Janssen | 2022 | ||
PREOPANC-1 / PREOPANC-2 NCT02172976 Dutch trials testing whether treating before surgery beats operating first. Chemoradiation first helped in the long run; FOLFIRINOX first did not clearly beat surgery-first with adjuvant chemotherapy. | FOLFIRINOX / mFOLFIRINOX | Pancreatic ductal adenocarcinoma | 2022 | |||
CheckMate 577 NCT02743494 A year of immunotherapy after surgery doubled the time before cancer returned in patients whose tumour had not fully responded to chemoradiation, though the survival gain did not reach significance. | Nivolumab | Oesophageal cancer, Gastric & gastro-oesophageal junction cancer | BMS | 2021 | ||
Interfant-06 NCT00550992 The largest infant leukaemia trial showed that intensifying chemotherapy did not help, and set the stage for adding blinatumomab instead. | Blinatumomab | Acute lymphoblastic leukaemia | 2019 | |||
POLO NCT02184195 The first biomarker-directed drug approval in pancreatic cancer, for the roughly 5-7% with inherited BRCA mutations, though it did not extend overall survival. | Olaparib | Pancreatic ductal adenocarcinoma | AstraZeneca, Merck | 2019 | ||
PRIMA / ENGOT-OV26 NCT02655016 Extended PARP maintenance to women without BRCA mutations, but the survival benefit did not materialise on longer follow-up. | Niraparib | Ovarian cancer | GSK (Tesaro) | 2019 | ||
IMpassion130 NCT02425891 IMpassion130 was the first immunotherapy success in breast cancer, later undermined when a sister trial failed and the approval was withdrawn. | Atezolizumab, Paclitaxel / nab-paclitaxel | Triple-negative breast cancer | Roche | 2018 | ||
BILCAP Six months of oral chemotherapy after surgery became the standard for bile duct cancer despite a technically negative primary result. | — | Biliary tract cancer | Cancer Research UK | 2017 | ||
CONVERT NCT00433563 Settled the radiotherapy schedule debate in limited-stage disease: neither schedule was superior, so twice-daily 45 Gy remains standard and once-daily is an acceptable alternative. | — | Small-cell lung cancer | Cancer Research UK, EORTC | 2017 | ||
SIOPEN HR-NBL1 NCT01704716 Europe's long-running high-risk neuroblastoma trial set busulfan-melphalan as the transplant regimen and showed that adding IL-2 to anti-GD2 therapy added toxicity but not benefit. | Dinutuximab (ch14.18) / dinutuximab beta | Neuroblastoma | , Children's Cancer and Leukaemia Group | 2017 | ||
PALOMA-2 NCT01740427 The trial that made palbociclib the first CDK4/6 inhibitor in routine use. It doubled progression-free time but did not extend life. | Palbociclib | HR-positive / HER2-negative breast cancer | Pfizer | 2016 | ||
ROMANA 1 and ROMANA 2 NCT01387269 ROMANA 1 and 2 were two phase 3 trials of an appetite-hormone mimic in lung cancer patients with wasting. Patients gained muscle but not grip strength, which split regulators: approved in Japan, rejected in Europe. | — | Non-small-cell lung cancer | 2016 | |||
GOG-0218 & ICON7 (bevacizumab) NCT00262847 Adding the blood-vessel blocker bevacizumab to first-line chemotherapy delayed relapse by a few months but did not extend life overall. | Bevacizumab, Carboplatin, Paclitaxel / nab-paclitaxel | Ovarian cancer | 2011 | |||
AMPLIFY-7P NCT05726864 The randomised test of an off-the-shelf KRAS vaccine after pancreatic surgery. It missed its primary goal in June 2026. | ELI-002 7P | Pancreatic ductal adenocarcinoma | Elicio Therapeutics | 2026 | ||
DREAM3R NCT04334759 The phase 3 test of durvalumab with chemotherapy was stopped early and did not meet its goal, despite an encouraging earlier study. | Durvalumab, Pemetrexed | Mesothelioma | 2026 | |||
Fianlimab + cemiplimab phase 3 (first-line melanoma) NCT05352672 A second LAG-3 blocker failed to beat pembrolizumab alone, a warning that the Opdualag result is not automatically a class effect. | Fianlimab, Cemiplimab, Pembrolizumab | Melanoma | Regeneron | 2026 | ||
LITESPARK-012 NCT04736706 Adding belzutifan to first-line lenvatinib-pembrolizumab did not deliver, a reminder that later-line success does not guarantee first-line benefit. | Belzutifan, Lenvatinib, Pembrolizumab | Renal cell carcinoma | Merck | 2026 | ||
persevERA NCT04546009 An oral SERD did not beat the aromatase inhibitor in first-line treatment when both were combined with palbociclib. Oral SERDs earn their place after resistance, not before it. | Giredestrant, Palbociclib | HR-positive / HER2-negative breast cancer | Roche, Genentech | 2026 | ||
XPORT-EC-042 / ENGOT-EN20 / GOG-3083 NCT05611931 A promising subgroup finding from an earlier trial did not hold up: selinexor maintenance missed its main goal in TP53-normal endometrial cancer. | Selinexor | Endometrial cancer | 2026 | |||
RELATIVITY-098 NCT05002569 Adding relatlimab to adjuvant nivolumab did not reduce recurrence, so the LAG-3 combination stays a treatment for measurable disease. | Relatlimab + nivolumab, Nivolumab | Melanoma | BMS | 2025 | ||
MARS 2 NCT02040272 MARS 2 is the trial that overturned decades of surgical practice: removing the lining of the lung did not help patients live longer and left them worse off. | — | Mesothelioma | 2024 | |||
NALIRICC (AIO) Adding a liposomal chemotherapy did not help in second-line bile duct cancer, contradicting an earlier Korean trial. | — | Biliary tract cancer | 2024 | |||
TiNivo-2 NCT04987203 A second trial confirming that restarting immunotherapy after it fails does not help kidney cancer patients. | Tivozanib, Nivolumab | Renal cell carcinoma | 2024 | |||
TrilynX NCT04459715 A promising phase 2 drug that sensitises tumours to radiation made things worse in phase 3, a reminder that early wins in this disease often do not replicate. | — | Head and neck squamous cell carcinoma | 2024 | |||
CONTACT-03 NCT04338269 Continuing immunotherapy after it has failed, alongside a targeted pill, added nothing but side effects. The first definitive test of 'IO rechallenge'. | Atezolizumab, Cabozantinib | Renal cell carcinoma | Roche, Exelixis | 2023 | ||
IMbrave050 NCT04102098 The first adjuvant immunotherapy trial in liver cancer looked positive early, then the benefit vanished with longer follow-up. | Atezolizumab | Hepatocellular carcinoma | Roche | 2023 | ||
LEAP-002 NCT03713593 Adding pembrolizumab to lenvatinib did not significantly extend life, a reminder that not every immunotherapy plus anti-angiogenic pairing works. | Lenvatinib, Pembrolizumab | Hepatocellular carcinoma | Merck, Eisai | 2022 | ||
ATLANTIS NCT02566993 Lurbinectedin's confirmatory trial missed its survival goal, but the drug stayed on the market because the combination tested was not the approved monotherapy dose. | Lurbinectedin, Topotecan | Small-cell lung cancer | PharmaMar, Jazz | 2021 | ||
BELINDA NCT03570892 The one second-line CAR-T trial that failed, a reminder that manufacturing time and trial design can erase a real effect. | — | Diffuse large B-cell lymphoma | Novartis | 2021 | ||
OUTBACK / ANZGOG 0902 / GOG-0274 NCT01414608 Giving extra chemotherapy after chemoradiation added side effects and no survival benefit, so timing matters: chemotherapy helps before radiation (INTERLACE), not after. | Carboplatin, Paclitaxel / nab-paclitaxel | Cervical cancer | , NRG Oncology | 2021 | ||
UKCTOCS NCT00058032 The largest screening trial ever run for ovarian cancer found earlier detection but no reduction in deaths, so there is still no recommended screening. | — | Ovarian cancer | , , CRUK | 2021 | ||
CheckMate 548 & CheckMate 143 & CheckMate 498 NCT02667587 CheckMate 548, 143 and 498 were three large trials, all negative: the immunotherapy that transformed melanoma and lung cancer did nothing in glioblastoma. | Nivolumab, Bevacizumab, Temozolomide | Glioma & glioblastoma | BMS | 2020 | ||
ERGO2: ketogenic diet and fasting during re-irradiation of recurrent glioma NCT01754350 The only randomised trial of a ketogenic diet in brain tumours found no benefit. Patients could follow the diet and their ketone levels rose, but progression came just as quickly. | — | Glioma & glioblastoma | 2020 | |||
IMpassion131 NCT03125902 IMpassion131 was the sister trial to the first immunotherapy success in breast cancer. It failed, and the approval it was meant to confirm was withdrawn. | Atezolizumab, Paclitaxel / nab-paclitaxel, Pembrolizumab | Triple-negative breast cancer | Roche | 2020 | ||
JAVELIN Head and Neck 100 NCT02952586 Adding a PD-L1 blocker to curative chemoradiation did not help, the first of several such failures in head and neck cancer. | — | Head and neck squamous cell carcinoma | , Pfizer | 2020 | ||
PALLAS & PENELOPE-B NCT02513394 Two large trials found that adding palbociclib after surgery does not prevent recurrence, even though the same drug helps in metastatic disease. | Palbociclib | HR-positive / HER2-negative breast cancer | , Alliance, , Pfizer | 2020 | ||
VOYAGER NCT03465722 Avapritinib failed to beat regorafenib in unselected later-line GIST, confining it to the PDGFRA D842V niche. | Avapritinib | Sarcomas | 2020 | |||
ANNOUNCE NCT02451943 A PDGFRα antibody that had won accelerated approval on a small trial failed to improve survival in the confirmatory study and was withdrawn. | Doxorubicin | Sarcomas | Eli Lilly | 2019 | ||
ASTRA NCT01843062 Adding a MEK inhibitor to boost iodine uptake before ablation did not improve complete remission rates, cooling the 'redifferentiation for everyone' idea. | — | Thyroid cancer | AstraZeneca | 2019 | ||
LUME-Meso NCT01907100 In LUME-Meso, a promising phase 2 signal for the multi-kinase inhibitor nintedanib vanished in phase 3. | Nintedanib, Pemetrexed | Mesothelioma | Boehringer Ingelheim | 2019 | ||
ASPREE (ASPirin in Reducing Events in the Elderly) NCT01038583 In healthy older people, daily low-dose aspirin did not extend disability-free life and, unexpectedly, was linked with more cancer deaths. It changed guidelines against routine aspirin in the elderly. | — | — | 2018 | |||
LACC (Laparoscopic Approach to Cervical Cancer) NCT00614211 Keyhole surgery, assumed equivalent, turned out to be worse: more recurrences and more deaths than open surgery, reversing practice overnight. | — | Cervical cancer | 2018 | |||
VITAL (VITamin D and OmegA-3 TriaL) NCT01169259 The definitive test of whether vitamin D or fish-oil pills prevent cancer or heart disease in healthy adults. They did not. | — | — | 2018 | |||
SARAH and SIRveNIB NCT01482442 Two large trials found radioactive beads were gentler than sorafenib but did not help patients live longer. | Sorafenib | Hepatocellular carcinoma | 2017 | |||
ACT IV NCT01480479 A double-blind test of a promising glioblastoma vaccine that showed no benefit, and taught the field how misleading historical-control comparisons can be. | Rindopepimut | Glioma & glioblastoma | Celldex Therapeutics | 2016 | ||
WHEL (Women's Healthy Eating and Living) NCT00003787 Three thousand breast cancer survivors were coached for years to eat far more vegetables, fruit and fibre. They did, and it made no difference to recurrence or survival. | — | HR-positive / HER2-negative breast cancer, Triple-negative breast cancer | , NCI | 2007 |