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Clinical Trials in the USA / NCT04924075
Recruiting Phase 2

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)

NCT04924075 · tracked via the Priya Life Science USA tracker
Sponsor
Merck Sharp & Dohme LLC
Phase
Phase 2
Started
2021-08-12
Last updated
2026-09-03

Condition(s) studied

Pheochromocytoma/ParagangliomaPancreatic Neuroendocrine TumorVon Hippel-Lindau DiseaseAdvanced Gastrointestinal Stromal TumorHIF-2α Mutated Cancers

Investigational drug(s) / intervention(s)

Belzutifan

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

Study summary

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).

Eligibility

Sex
ALL
Min age
12 Years
Max age
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (84)

FacilityCityRegionStatus
Cedars-Sinai Medical Center ( Site 0110) Los Angeles California Recruiting
Northwestern University - Robert H. Lurie Comprehensive Cancer Center ( Site 0130) Chicago Illinois Recruiting
Northwestern Medicine Cancer Center - Warrenville ( Site 0134) Warrenville Illinois Recruiting
University of Iowa ( Site 0104) Iowa City Iowa Recruiting
Johns Hopkins Hospital-Sidney Kimmel Comprehensive Cancer Center - Developmental Therapeutics ( Site 0108) Baltimore Maryland Recruiting
National Institutes of Health ( Site 0125) Bethesda Maryland Recruiting
Massachusetts General Hospital ( Site 0111) Boston Massachusetts Recruiting
University of Michigan ( Site 0126) Ann Arbor Michigan Recruiting
Washington University-Internal Medicine/Oncology ( Site 0124) St Louis Missouri Active Not Recruiting
Icahn School of Medicine at Mount Sinai ( Site 0123) New York New York Active Not Recruiting
Penn Medicine: University of Pennsylvania Health System-Heme/Onc ( Site 0127) Philadelphia Pennsylvania Active Not Recruiting
SCRI Oncology Partners ( Site 7000) Nashville Tennessee Recruiting
Vanderbilt University Medical Center ( Site 0107) Nashville Tennessee Recruiting
University of Texas MD Anderson Cancer Center ( Site 0112) Houston Texas Recruiting
Prince of Wales Hospital-Medical Oncology ( Site 1601) Randwick New South Wales Recruiting
The Royal Melbourne Hospital ( Site 1602) Parkville Victoria Recruiting
Arthur J.E. Child Comprehensive Cancer Centre ( Site 0203) Calgary Alberta Recruiting
Princess Margaret Cancer Centre ( Site 0202) Toronto Ontario Recruiting
FALP ( Site 2200) Santiago Region M. de Santiago Recruiting
Centro de Oncología de Precisión ( Site 2203) Santiago Region M. de Santiago Recruiting
Peking University First Hospital-Urology ( Site 1900) Beijing Beijing Municipality Active Not Recruiting
Sun Yat-sen University Cancer Center ( Site 1905) Guangzhou Guangdong Active Not Recruiting
Renji Hospital Shanghai Jiao Tong University School of Medicine ( Site 1904) Shanghai Shanghai Municipality Active Not Recruiting
West China Hospital of Sichuan University ( Site 1906) Chengdu Sichuan Completed
Rigshospitalet ( Site 0304) Copenhagen Capital Region Recruiting
Rigshospitalet-Department of Endocrinology ( Site 0303) Copenhagen Capital Region Recruiting
Odense Universitetshospital ( Site 0302) Odense Region Syddanmark Recruiting
CHU Strasbourg-Hautepierre-Medecine Interne, Endocrinologie et Nutrition ( Site 0402) Strasbourg Alsace Recruiting
Hôpital Edouard Herriot-oncologie ( Site 0405) Lyon Auvergne-Rhône-Alpes Recruiting
Institut Paoli-Calmettes-Oncology ( Site 0406) Marseille Bouches-du-Rhone Active Not Recruiting
Hôpitaux Universitaires Paris Sud - Hôpital Bicêtre ( Site 0407) Le Kremlin-Bicêtre Paris Recruiting
Hopitaux Universitaires Paris Centre-Hopital Cochin ( Site 0404) Paris France Recruiting
Gustave Roussy ( Site 0403) Villejuif Île-de-France Region Active Not Recruiting
Universitaetsklinikum Freiburg ( Site 0504) Freiburg im Breisgau Baden-Wurttemberg Recruiting
Klinikum der Ludwig-Maximilians-Universitaet Muenchen-Department of Internal Medicine IV, Division ( Site 0501) München Bavaria Completed
Comprehensive Cancer Center Mainfranken-Div. of Endocrinology and Diabetes ( Site 0500) Würzburg Bavaria Recruiting
Universitaetsklinikum Duesseldorf-Gastroenterology, Hepatology and Infectiology ( Site 0505) Düsseldorf North Rhine-Westphalia Recruiting
Charité Universitaetsmedizin Berlin - Campus Mitte-Department of Endocrinology and Metabolism ( Site 0503) Berlin Germany Recruiting
Semmelweis University-Belgyógyászati és Onkológiai Klinika Hematológia Osztály ( Site 0600) Budapest Hungary Recruiting
Sheba Medical Center-Institute of Endocrinology, Diabetes and Metabolism ( Site 1400) Ramat Gan Israel Recruiting

+ 44 more sites — see the full list on the official registry below.

More Merck Sharp & Dohme LLC trials in the USA

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04924075 on ClinicalTrials.gov ↗ ← All trials in the USA