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Recruiting NCT04924075

Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)

Phase II Interventional Pheochromocytoma/Paraganglioma Pancreatic Neuroendocrine Tumor Von Hippel-Lindau Disease Advanced Gastrointestinal Stromal Tumor

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Belzutifan.
Who it may be relevant to
Registry conditions: Pheochromocytoma/Paraganglioma, Pancreatic Neuroendocrine Tumor, Von Hippel-Lindau Disease, Advanced Gastrointestinal Stromal Tumor. Basic parameters: from 12 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Australia, Canada, Chile, China +17
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 2 Study to Evaluate the Efficacy and Safety of Belzutifan (MK-6482, Formerly PT2977) Monotherapy in Participants With Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Advanced Solid Tumors With HIF-2α Related Genetic Alterations

Overview

This is a study to evaluate the efficacy and safety of belzutifan monotherapy in participants with advanced pheochromocytoma/paraganglioma (PPGL), pancreatic neuroendocrine tumor (pNET), von Hippel-Lindau (VHL) disease-associated tumors, advanced wt (wild-type) gastrointestinal stromal tumor (wt GIST), or advanced solid tumors with hypoxia inducible factor-2 alpha (HIF-2α) related genetic alterations. The primary objective of the study is to evaluate the objective response rate (ORR) of belzutifan per response evaluation criteria in solid tumors version 1.1 (RECIST 1.1) by blinded independent central review (BICR).

Interventions

  • Drug Belzutifan
    Belzutifan, 120 mg, oral, once daily (QD) until progressive disease or discontinuation.

Primary outcome measures

  • Objective Response Rate (ORR) as Assessed by Blinded Independent Central Review (BICR) [Time frame: Up to approximately 5.5 years]
Secondary outcome measures (8)
  • Duration of Response (DOR) as Assessed by BICR [Time frame: Up to approximately 5.5 years]
  • Time to Response (TTR) as Assessed by BICR [Time frame: Up to approximately 5.5 years]
  • Disease Control Rate (DCR) as Assessed by BICR [Time frame: Up to approximately 5.5 years]
  • Progressive Free Survival (PFS) as Assessed by BICR [Time frame: Up to approximately 5.5 years]
  • Overall Survival (OS) [Time frame: Up to approximately 5.5 years]
  • Number of Participants Who Experience an Adverse Event (AE) [Time frame: Up to approximately 5.5 years]
  • Number of Participants Who Discontinue Study Treatment Due to an AE [Time frame: Up to approximately 5.5 years]
  • Time to Surgery (TTS) [Time frame: Up to approximately 5.5 years]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 14 centers
  • Cedars-Sinai Medical Center ( Site 0110) — Los Angeles
  • Northwestern University - Robert H. Lurie Comprehensive Cancer Center ( Site 0130) — Chicago
  • Northwestern Medicine Cancer Center - Warrenville ( Site 0134) — Warrenville
  • University of Iowa ( Site 0104) — Iowa City
  • Johns Hopkins Hospital-Sidney Kimmel Comprehensive Cancer Center - Developmental Therapeut — Baltimore
  • National Institutes of Health ( Site 0125) — Bethesda
  • Massachusetts General Hospital ( Site 0111) — Boston
  • University of Michigan ( Site 0126) — Ann Arbor
  • … and 6 more centers
Italy · 8 centers
  • University of Naples Federico II-Dipartimento di Medicina Clinica e Chirurgia ( Site 0704) — Naples
  • Fondazione IRCCS Istituto Nazionale dei Tumori-S.C. Oncologia Medica 2- Sarcomi ( Site 070 — Milan
  • Azienda Ospedaliero Universitaria Senese-U.O.C. Immunoterapia Oncologica ( Site 0706) — Siena
  • IRCCS Azienda Ospedaliero-Universitaria di Bologna, Policlinico di Sant'Orsola ( Site 0708 — Bologna
  • Azienda Ospedaliera Spedali Civili di Brescia-Oncology ( Site 0701) — Brescia
  • Ospedale San Raffaele-Oncologia Medica ( Site 0705) — Milan
  • Istituto Europeo di Oncologia IRCCS-Divisione di Oncologia Medica Gastrointestinale e Tumo — Milan
  • Centro Ricerche Cliniche di Verona ( Site 0703) — Verona
France · 6 centers
  • CHU Strasbourg-Hautepierre-Medecine Interne, Endocrinologie et Nutrition ( Site 0402) — Strasbourg
  • Hôpital Edouard Herriot-oncologie ( Site 0405) — Lyon
  • Institut Paoli-Calmettes-Oncology ( Site 0406) — Marseille
  • Hôpitaux Universitaires Paris Sud - Hôpital Bicêtre ( Site 0407) — Le Kremlin-Bicêtre
  • Hopitaux Universitaires Paris Centre-Hopital Cochin ( Site 0404) — Paris
  • Gustave Roussy ( Site 0403) — Villejuif
Japan · 6 centers
  • Hokkaido University Hospital ( Site 1800) — Sapporo
  • Yokohama City University Hospital ( Site 1804) — Yokohama
  • Kochi Medical School Hospital ( Site 1807) — Nankoku
  • National Cancer Center Hospital ( Site 1802) — Chuo-ku
  • Kyoto University Hospital ( Site 1806) — Kyoto
  • Tokyo Women's Medical University Adachi Medical Center ( Site 1803) — Tokyo
Germany · 5 centers
  • Universitaetsklinikum Freiburg ( Site 0504) — Freiburg im Breisgau
  • Klinikum der Ludwig-Maximilians-Universitaet Muenchen-Department of Internal Medicine IV, — München
  • Comprehensive Cancer Center Mainfranken-Div. of Endocrinology and Diabetes ( Site 0500) — Würzburg
  • Universitaetsklinikum Duesseldorf-Gastroenterology, Hepatology and Infectiology ( Site 050 — Düsseldorf
  • Charité Universitaetsmedizin Berlin - Campus Mitte-Department of Endocrinology and Metabol — Berlin
Russia · 5 centers
  • GBUZ Republican Clinical Oncological Dispensary ( Site 0804) — Ufa
  • Saint Petersburg State University-Clinic of advanced medical technologies n. a. Nicolay I. — Saint Petersburg
  • Saint-Petersburg City Clinical Oncology Dispensary-Department of chemotherapy ( Site 0803) — Saint Petersburg
  • Fed State Budgetary Inst N.N. Blokhin Med Center of Oncology MHRF ( Site 0801) — Moscow
  • Endocrinology Research Center of Rosmedtechnologies-Surgery ( Site 0809) — Moscow
Turkey (Türkiye) · 5 centers
  • Baskent University Adana Training Hospital ( Site 0906) — Yüreğir
  • Ege University Medicine of Faculty ( Site 0900) — Bornova
  • Hacettepe Universitesi-oncology hospital ( Site 0901) — Ankara
  • Ankara Bilkent Şehir Hastanesi. ( Site 0904) — Ankara
  • Istanbul Universitesi Cerrahpasa-Medical Oncology ( Site 0902) — Istanbul
China · 4 centers
  • Peking University First Hospital-Urology ( Site 1900) — Beijing
  • Sun Yat-sen University Cancer Center ( Site 1905) — Guangzhou
  • Renji Hospital Shanghai Jiao Tong University School of Medicine ( Site 1904) — Shanghai
  • West China Hospital of Sichuan University ( Site 1906) — Chengdu
Spain · 4 centers
  • MD Anderson Cancer Center-Oncology ( Site 1102) — Madrid
  • Hospital Universitario 12 de Octubre-Medical Oncology ( Site 1103) — Madrid
  • Hospital Universitario Central de Asturias-Medical Oncology ( Site 1101) — Oviedo
  • Hospital Universitari Vall d'Hebron ( Site 1100) — Barcelona
Sweden · 4 centers
  • Skanes University Hospital Lund ( Site 1200) — Lund
  • Karolinska Universitetssjukhuset Solna ( Site 1202) — Stockholm
  • Akademiska sjukhuset-Blod- och tumörsjukdomar ( Site 1201) — Uppsala
  • Sahlgrenska Universitetssjukhuset-Department of Oncology CTU Clinical Trial Unit ( Site 12 — Gothenburg
United Kingdom · 4 centers

Center list to be confirmed — check the primary protocol.

Denmark · 3 centers
  • Rigshospitalet ( Site 0304) — Copenhagen
  • Rigshospitalet-Department of Endocrinology ( Site 0303) — Copenhagen
  • Odense Universitetshospital ( Site 0302) — Odense
Australia · 2 centers
  • Prince of Wales Hospital-Medical Oncology ( Site 1601) — Randwick
  • The Royal Melbourne Hospital ( Site 1602) — Parkville
Canada · 2 centers
  • Arthur J.E. Child Comprehensive Cancer Centre ( Site 0203) — Calgary
  • Princess Margaret Cancer Centre ( Site 0202) — Toronto
Chile · 2 centers
  • FALP ( Site 2200) — Santiago
  • Centro de Oncología de Precisión ( Site 2203) — Santiago
Israel · 2 centers
  • Sheba Medical Center-Institute of Endocrinology, Diabetes and Metabolism ( Site 1400) — Ramat Gan
  • Sourasky Medical Center ( Site 1401) — Tel Aviv
Portugal · 2 centers
  • START Lisbon - Hospital de Santa Maria ( Site 2601) — Lisbon
  • Instituto Portugues de Oncologia Do Porto Francisco Gentil E.P.E. ( Site 2600) — Porto
South Korea · 2 centers
  • Seoul National University Hospital ( Site 2001) — Jongno-gu
  • Asan Medical Center ( Site 2000) — Songpa-gu
Hungary · 1 center
  • Semmelweis University-Belgyógyászati és Onkológiai Klinika Hematológia Osztály ( Site 0600 — Budapest
Netherlands · 1 center
  • Universitair Medisch Centrum Utrecht ( Site 1530) — Utrecht
Norway · 1 center
  • Oslo Universitetssykehus Radiumhospitalet ( Site 2400) — Oslo
Singapore · 1 center
  • National Cancer Centre Singapore ( Site 1700) — Singapore

Publications

  • Jimenez C, Andreassen M, Durand A, Moog S, Hendifar A, Welin S, Spada F, Sharma R, Wolin E, Ruether J, Garcia-Carbonero R, Fassnacht M, Capdevila J, Del Rivero J, Iliopoulos O, Huillard O, Jang R, Mai K, Artamonova E, Hallqvist A, Else T, Odeleye-Ajakaye A, Gozman A, Naik GS, Berruti A; LITESPARK-015 Investigators. Belzutifan for Advanced Pheochromocytoma or Paraganglioma. N Engl J Med. 2025 Nov 2 PMID 41124218

Identifiers

NCT: NCT04924075 · 6482-015 · MK-6482-015 · PT2977 · jRCT2011220024 · 2023-504853-11-00 · 2020-005028-13

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗