New pill shows promise for rare tumors in Mid-Stage trial

NCT ID NCT04924075

Recruiting now Disease control Sponsor: Merck Sharp & Dohme LLC Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 04, 2026 · Updated 6 times

Summary

This phase 2 study tests belzutifan, a daily pill that blocks a protein called HIF-2α, in people with advanced rare cancers like pheochromocytoma/paraganglioma, pancreatic neuroendocrine tumors, VHL disease-associated tumors, and certain other solid tumors. The goal is to see if the drug can shrink tumors or stop them from growing. About 355 participants aged 12 and older are being enrolled at multiple sites.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: belzutifan (a pill that blocks a protein called HIF-2α to slow tumor growth)
What this could lead to: If successful, belzutifan could offer a new treatment option for several rare and hard-to-treat cancers, potentially shrinking tumors or slowing their growth.
What could go wrong: This is a mid-stage trial with no control group, so results are preliminary. The drug may not work for all tumor types, and side effects are still being studied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

Adenoma, Islet Cell gastrointestinal stromal tumor pancreatic neuroendocrine tumor paraganglioma pheochromocytoma pheochromocytoma-paraganglioma von Hippel-Lindau disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ADVANCED GASTROINTESTINAL STROMAL TUMOR HIF-2Α MUTATED CANCERS PANCREATIC NEUROENDOCRINE TUMOR PHEOCHROMOCYTOMA/PARAGANGLIOMA VON HIPPEL-LINDAU DISEASE

Contacts and locations

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Study contacts

Contact

Email: •••••@•••••

Locations

Addenbrooke's Hospital ( Site 1309)
RECRUITING

Cambridge, Cambridgeshire, CB2 2QQ, United Kingdom
Akademiska sjukhuset-Blod- och tumörsjukdomar ( Site 1201)
RECRUITING

Uppsala, Uppsala County, 751 85, Sweden
Ankara Bilkent Şehir Hastanesi. ( Site 0904)
RECRUITING

Ankara, 06800, Turkey (Türkiye)
Arthur J.E. Child Comprehensive Cancer Centre ( Site 0203)
RECRUITING

Calgary, Alberta, T2N 5G2, Canada
Asan Medical Center ( Site 2000)
RECRUITING

Songpa-gu, Seoul, 05505, South Korea
Azienda Ospedaliera Spedali Civili di Brescia-Oncology ( Site 0701)
RECRUITING

Brescia, 25123, Italy
Azienda Ospedaliero Universitaria Senese-U.O.C. Immunoterapia Oncologica ( Site 0706)
RECRUITING

Siena, Tuscany, 53100, Italy
Baskent University Adana Training Hospital ( Site 0906)
RECRUITING

Yüreğir, Adana, 01250, Turkey (Türkiye)
CHU Strasbourg-Hautepierre-Medecine Interne, Endocrinologie et Nutrition ( Site 0402)
RECRUITING

Strasbourg, Alsace, 67098, France
Cedars-Sinai Medical Center ( Site 0110)
RECRUITING

Los Angeles, California, 90048, United States
Centro Ricerche Cliniche di Verona ( Site 0703)
RECRUITING

Verona, 37134, Italy
Centro de Oncología de Precisión ( Site 2203)
RECRUITING

Santiago, Region M. de Santiago, 7560908, Chile
Charité Universitaetsmedizin Berlin - Campus Mitte-Department of Endocrinology and Metabolism ( Site 0503)
RECRUITING

Berlin, 10117, Germany
Comprehensive Cancer Center Mainfranken-Div. of Endocrinology and Diabetes ( Site 0500)
RECRUITING

Würzburg, Bavaria, 97080, Germany
Ege University Medicine of Faculty ( Site 0900)
RECRUITING

Bornova, İzmir, 35100, Turkey (Türkiye)
Endocrinology Research Center of Rosmedtechnologies-Surgery ( Site 0809)
COMPLETED

Moscow, Moscow, 117036, Russia
FALP ( Site 2200)
RECRUITING

Santiago, Region M. de Santiago, 7500921, Chile
Fed State Budgetary Inst N.N. Blokhin Med Center of Oncology MHRF ( Site 0801)
ACTIVE_NOT_RECRUITING

Moscow, Moscow, 115522, Russia
Fondazione IRCCS Istituto Nazionale dei Tumori-S.C. Oncologia Medica 2- Sarcomi ( Site 0709)
RECRUITING

Milan, Lombardy, 20133, Italy
GBUZ Republican Clinical Oncological Dispensary ( Site 0804)
COMPLETED

Ufa, Baskortostan, Respublika, 450054, Russia
Gustave Roussy ( Site 0403)
ACTIVE_NOT_RECRUITING

Villejuif, Île-de-France Region, 94805, France
Hacettepe Universitesi-oncology hospital ( Site 0901)
RECRUITING

Ankara, 06230, Turkey (Türkiye)
Hammersmith Hospital-Medical Oncology ( Site 1304)
RECRUITING

London, London, City of, W12 OHS, United Kingdom
Hokkaido University Hospital ( Site 1800)
ACTIVE_NOT_RECRUITING

Sapporo, Hokkaido, 060-8648, Japan
Hopitaux Universitaires Paris Centre-Hopital Cochin ( Site 0404)
RECRUITING

Paris, 75014, France
Hospital Universitari Vall d'Hebron ( Site 1100)
RECRUITING

Barcelona, 08035, Spain
Hospital Universitario 12 de Octubre-Medical Oncology ( Site 1103)
RECRUITING

Madrid, Madrid, Comunidad de, 28041, Spain
Hospital Universitario Central de Asturias-Medical Oncology ( Site 1101)
RECRUITING

Oviedo, Principality of Asturias, 33011, Spain
Hôpital Edouard Herriot-oncologie ( Site 0405)
RECRUITING

Lyon, Auvergne-Rhône-Alpes, 69003, France
Hôpitaux Universitaires Paris Sud - Hôpital Bicêtre ( Site 0407)
RECRUITING

Le Kremlin-Bicêtre, Paris, 94270, France
IRCCS Azienda Ospedaliero-Universitaria di Bologna, Policlinico di Sant'Orsola ( Site 0708)
RECRUITING

Bologna, 40138, Italy
Icahn School of Medicine at Mount Sinai ( Site 0123)
ACTIVE_NOT_RECRUITING

New York, New York, 10029, United States
Institut Paoli-Calmettes-Oncology ( Site 0406)
ACTIVE_NOT_RECRUITING

Marseille, Bouches-du-Rhone, 13009, France
Instituto Portugues de Oncologia Do Porto Francisco Gentil E.P.E. ( Site 2600)
RECRUITING

Porto, 4200-072, Portugal
Istanbul Universitesi Cerrahpasa-Medical Oncology ( Site 0902)
COMPLETED

Istanbul, 34668, Turkey (Türkiye)
Istituto Europeo di Oncologia IRCCS-Divisione di Oncologia Medica Gastrointestinale e Tumori Neuroe ( Site 0700)
RECRUITING

Milan, 20141, Italy
Johns Hopkins Hospital-Sidney Kimmel Comprehensive Cancer Center - Developmental Therapeutics ( Site 0108)
RECRUITING

Baltimore, Maryland, 21287, United States
Karolinska Universitetssjukhuset Solna ( Site 1202)
COMPLETED

Stockholm, Stockholm County, 171 76, Sweden
Klinikum der Ludwig-Maximilians-Universitaet Muenchen-Department of Internal Medicine IV, Division ( Site 0501)
COMPLETED

München, Bavaria, 80336, Germany
Kochi Medical School Hospital ( Site 1807)
ACTIVE_NOT_RECRUITING

Nankoku, Kochi, 783-8505, Japan
Kyoto University Hospital ( Site 1806)
ACTIVE_NOT_RECRUITING

Kyoto, 606-8507, Japan
MD Anderson Cancer Center-Oncology ( Site 1102)
COMPLETED

Madrid, Madrid, Comunidad de, 28033, Spain
Massachusetts General Hospital ( Site 0111)
RECRUITING

Boston, Massachusetts, 02114, United States
National Cancer Center Hospital ( Site 1802)
ACTIVE_NOT_RECRUITING

Chuo-ku, Tokyo, 104-0045, Japan
National Cancer Centre Singapore ( Site 1700)
RECRUITING

Singapore, Central Singapore, 168583, Singapore
National Institutes of Health ( Site 0125)
RECRUITING

Bethesda, Maryland, 20892, United States
Northwestern Medicine Cancer Center - Warrenville ( Site 0134)
RECRUITING

Warrenville, Illinois, 60555, United States
Northwestern University - Robert H. Lurie Comprehensive Cancer Center ( Site 0130)
RECRUITING

Chicago, Illinois, 60611, United States
Odense Universitetshospital ( Site 0302)
RECRUITING

Odense, Region Syddanmark, 5000, Denmark
Oslo Universitetssykehus Radiumhospitalet ( Site 2400)
RECRUITING

Oslo, 0424, Norway
Ospedale San Raffaele-Oncologia Medica ( Site 0705)
COMPLETED

Milan, 20132, Italy
Peking University First Hospital-Urology ( Site 1900)
ACTIVE_NOT_RECRUITING

Beijing, Beijing Municipality, 100034, China
Penn Medicine: University of Pennsylvania Health System-Heme/Onc ( Site 0127)
ACTIVE_NOT_RECRUITING

Philadelphia, Pennsylvania, 19104, United States
Prince of Wales Hospital-Medical Oncology ( Site 1601)
RECRUITING

Randwick, New South Wales, 2031, Australia
Princess Margaret Cancer Centre ( Site 0202)
RECRUITING

Toronto, Ontario, M5G 2M9, Canada
Renji Hospital Shanghai Jiao Tong University School of Medicine ( Site 1904)
ACTIVE_NOT_RECRUITING

Shanghai, Shanghai Municipality, 200001, China
Rigshospitalet ( Site 0304)
RECRUITING

Copenhagen, Capital Region, 2100, Denmark
Rigshospitalet-Department of Endocrinology ( Site 0303)
RECRUITING

Copenhagen, Capital Region, 2100, Denmark
Royal Free Hospital ( Site 1302)
ACTIVE_NOT_RECRUITING

London, England, NW3 2QG, United Kingdom
SCRI Oncology Partners ( Site 7000)
RECRUITING

Nashville, Tennessee, 37203, United States
START Lisbon - Hospital de Santa Maria ( Site 2601)
RECRUITING

Lisbon, Lisbon District, 1649-035, Portugal
Sahlgrenska Universitetssjukhuset-Department of Oncology CTU Clinical Trial Unit ( Site 1204)
RECRUITING

Gothenburg, Västra Götaland County, 413 45, Sweden
Saint Petersburg State University-Clinic of advanced medical technologies n. a. Nicolay I. Pirogov ( Site 0805)
COMPLETED

Saint Petersburg, Leningradskaya Oblast', 190020, Russia
Saint-Petersburg City Clinical Oncology Dispensary-Department of chemotherapy ( Site 0803)
ACTIVE_NOT_RECRUITING

Saint Petersburg, Leningradskaya Oblast', 198255, Russia
Semmelweis University-Belgyógyászati és Onkológiai Klinika Hematológia Osztály ( Site 0600)
RECRUITING

Budapest, 1083, Hungary
Seoul National University Hospital ( Site 2001)
RECRUITING

Jongno-gu, Seoul, 03080, South Korea
Sheba Medical Center-Institute of Endocrinology, Diabetes and Metabolism ( Site 1400)
RECRUITING

Ramat Gan, 5262100, Israel
Skanes University Hospital Lund ( Site 1200)
RECRUITING

Lund, Skåne County, 221 85, Sweden
Sourasky Medical Center ( Site 1401)
RECRUITING

Tel Aviv, 6423906, Israel
Sun Yat-sen University Cancer Center ( Site 1905)
ACTIVE_NOT_RECRUITING

Guangzhou, Guangdong, 510060, China
The Beatson West of Scotland Cancer Centre ( Site 1308)
RECRUITING

Glasgow, Glasgow City, G12 0YN, United Kingdom
The Royal Melbourne Hospital ( Site 1602)
RECRUITING

Parkville, Victoria, 3050, Australia
Tokyo Women's Medical University Adachi Medical Center ( Site 1803)
ACTIVE_NOT_RECRUITING

Tokyo, 123-8558, Japan
Universitaetsklinikum Duesseldorf-Gastroenterology, Hepatology and Infectiology ( Site 0505)
RECRUITING

Düsseldorf, North Rhine-Westphalia, 40225, Germany
Universitaetsklinikum Freiburg ( Site 0504)
RECRUITING

Freiburg im Breisgau, Baden-Wurttemberg, 79106, Germany
Universitair Medisch Centrum Utrecht ( Site 1530)
RECRUITING

Utrecht, 3584 CX, Netherlands
University of Iowa ( Site 0104)
RECRUITING

Iowa City, Iowa, 52242, United States
University of Michigan ( Site 0126)
RECRUITING

Ann Arbor, Michigan, 48109, United States
University of Naples Federico II-Dipartimento di Medicina Clinica e Chirurgia ( Site 0704)
COMPLETED

Naples, Campania, 80100, Italy
University of Texas MD Anderson Cancer Center ( Site 0112)
RECRUITING

Houston, Texas, 77030, United States
Vanderbilt University Medical Center ( Site 0107)
RECRUITING

Nashville, Tennessee, 37232, United States
Washington University-Internal Medicine/Oncology ( Site 0124)
ACTIVE_NOT_RECRUITING

St Louis, Missouri, 63110, United States
West China Hospital of Sichuan University ( Site 1906)
COMPLETED

Chengdu, Sichuan, 610041, China
Yokohama City University Hospital ( Site 1804)
ACTIVE_NOT_RECRUITING

Yokohama, Kanagawa, 236-0004, Japan

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