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

NCT ID NCT04924075

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 8 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.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 355 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2021

Expected to finish

Dec 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

The main inclusion criteria include but are not limited to the following: * Male and female participants at least 12 years of age (at least 18 years of age for Cohort B1) * Diagnosis of one of the following: Advanced/metastatic pheochromocytoma/paraganglioma (PPGL), pancreatic neuroendocrine tumors (pNET), von Hippel-Lindau (VHL) disease associated localized tumors, or advanced wild-type gastrointestinal stromal tumor (wt GIST) or advanced solid tumors with Hypoxia Inducible Factor- 2 alpha subunit (HIF-2α) related genetic alterations * Cohort B1: VHL Disease-associated tumors: * Have a diagnosis of VHL disease as determined by a germline test locally and/or clinical diagnosis * Must be ≥18 years of age * Has a life expectancy of at least 3 months The main exclusion criteria include but are not limited to the following: * Unable to swallow orally administered medication or has a disorder that might affect the absorption of belzutifan * History of a second malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years * Any of the following: A pulse oximeter reading \<92% at rest, or requires intermittent supplemental oxygen, or requires chronic supplemental oxygen * Clinically significant cardiac disease, including unstable angina, acute myocardial infarction, or arterial bypass (CABG) or Percutaneous transluminal coronary angioplasty (PTCA) ≤6 months from study entry, or New York Heart Association Class III or IV congestive heart failure * Received prior treatment (except somatostatin analogs) with chemotherapy, targeted therapy, biologics, or other investigational therapy within the past 4 weeks of first dose of study intervention

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Get notified about this study

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    56 sites in 18 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

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)

    ACTIVE_NOT_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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