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)

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).

Interventions

• Belzutifan · DRUG

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

Published eligibility criteria

Verbatim from the registry. Only the site can decide how these apply to a specific patient.

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

Sites (84)

• 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, Australia · RECRUITING
• The Royal Melbourne Hospital ( Site 1602), Parkville, Victoria, Australia · RECRUITING
• Arthur J.E. Child Comprehensive Cancer Centre ( Site 0203), Calgary, Alberta, Canada · RECRUITING
• Princess Margaret Cancer Centre ( Site 0202), Toronto, Ontario, Canada · RECRUITING
• FALP ( Site 2200), Santiago, Region M. de Santiago, Chile · RECRUITING
• Centro de Oncología de Precisión ( Site 2203), Santiago, Region M. de Santiago, Chile · RECRUITING
• Peking University First Hospital-Urology ( Site 1900), Beijing, Beijing Municipality, China · ACTIVE_NOT_RECRUITING
• Sun Yat-sen University Cancer Center ( Site 1905), Guangzhou, Guangdong, China · ACTIVE_NOT_RECRUITING
• Renji Hospital Shanghai Jiao Tong University School of Medicine ( Site 1904), Shanghai, Shanghai Municipality, China · ACTIVE_NOT_RECRUITING
• West China Hospital of Sichuan University ( Site 1906), Chengdu, Sichuan, China · COMPLETED
• Rigshospitalet ( Site 0304), Copenhagen, Capital Region, Denmark · RECRUITING
• Rigshospitalet-Department of Endocrinology ( Site 0303), Copenhagen, Capital Region, Denmark · RECRUITING
• Odense Universitetshospital ( Site 0302), Odense, Region Syddanmark, Denmark · RECRUITING
• CHU Strasbourg-Hautepierre-Medecine Interne, Endocrinologie et Nutrition ( Site 0402), Strasbourg, Alsace, France · RECRUITING
• Hôpital Edouard Herriot-oncologie ( Site 0405), Lyon, Auvergne-Rhône-Alpes, France · RECRUITING
• Institut Paoli-Calmettes-Oncology ( Site 0406), Marseille, Bouches-du-Rhone, France · ACTIVE_NOT_RECRUITING
• Hôpitaux Universitaires Paris Sud - Hôpital Bicêtre ( Site 0407), Le Kremlin-Bicêtre, Paris, France · RECRUITING
• Hopitaux Universitaires Paris Centre-Hopital Cochin ( Site 0404), Paris, France · RECRUITING
• Gustave Roussy ( Site 0403), Villejuif, Île-de-France Region, France · ACTIVE_NOT_RECRUITING
• Universitaetsklinikum Freiburg ( Site 0504), Freiburg im Breisgau, Baden-Wurttemberg, Germany · RECRUITING
• Klinikum der Ludwig-Maximilians-Universitaet Muenchen-Department of Internal Medicine IV, Division ( Site 0501), München, Bavaria, Germany · COMPLETED
• Comprehensive Cancer Center Mainfranken-Div. of Endocrinology and Diabetes ( Site 0500), Würzburg, Bavaria, Germany · RECRUITING
• Universitaetsklinikum Duesseldorf-Gastroenterology, Hepatology and Infectiology ( Site 0505), Düsseldorf, North Rhine-Westphalia, Germany · 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
• Sourasky Medical Center ( Site 1401), Tel Aviv, Israel · RECRUITING
• University of Naples Federico II-Dipartimento di Medicina Clinica e Chirurgia ( Site 0704), Naples, Campania, Italy · COMPLETED
• Fondazione IRCCS Istituto Nazionale dei Tumori-S.C. Oncologia Medica 2- Sarcomi ( Site 0709), Milan, Lombardy, Italy · RECRUITING
• Azienda Ospedaliero Universitaria Senese-U.O.C. Immunoterapia Oncologica ( Site 0706), Siena, Tuscany, Italy · RECRUITING
• IRCCS Azienda Ospedaliero-Universitaria di Bologna, Policlinico di Sant'Orsola ( Site 0708), Bologna, Italy · RECRUITING
• Azienda Ospedaliera Spedali Civili di Brescia-Oncology ( Site 0701), Brescia, Italy · RECRUITING
• Ospedale San Raffaele-Oncologia Medica ( Site 0705), Milan, Italy · COMPLETED
• Istituto Europeo di Oncologia IRCCS-Divisione di Oncologia Medica Gastrointestinale e Tumori Neuroe ( Site 0700), Milan, Italy · RECRUITING
• Centro Ricerche Cliniche di Verona ( Site 0703), Verona, Italy · RECRUITING
• Hokkaido University Hospital ( Site 1800), Sapporo, Hokkaido, Japan · ACTIVE_NOT_RECRUITING
• Yokohama City University Hospital ( Site 1804), Yokohama, Kanagawa, Japan · ACTIVE_NOT_RECRUITING
• Kochi Medical School Hospital ( Site 1807), Nankoku, Kochi, Japan · ACTIVE_NOT_RECRUITING
• National Cancer Center Hospital ( Site 1802), Chuo-ku, Tokyo, Japan · ACTIVE_NOT_RECRUITING
• Kyoto University Hospital ( Site 1806), Kyoto, Japan · ACTIVE_NOT_RECRUITING
• Tokyo Women's Medical University Adachi Medical Center ( Site 1803), Tokyo, Japan · ACTIVE_NOT_RECRUITING
• Universitair Medisch Centrum Utrecht ( Site 1530), Utrecht, Netherlands · RECRUITING
• Oslo Universitetssykehus Radiumhospitalet ( Site 2400), Oslo, Norway · RECRUITING
• START Lisbon - Hospital de Santa Maria ( Site 2601), Lisbon, Lisbon District, Portugal · RECRUITING
• Instituto Portugues de Oncologia Do Porto Francisco Gentil E.P.E. ( Site 2600), Porto, Portugal · RECRUITING
• GBUZ Republican Clinical Oncological Dispensary ( Site 0804), Ufa, Baskortostan, Respublika, Russia · COMPLETED
• Saint Petersburg State University-Clinic of advanced medical technologies n. a. Nicolay I. Pirogov ( Site 0805), Saint Petersburg, Leningradskaya Oblast', Russia · COMPLETED
• Saint-Petersburg City Clinical Oncology Dispensary-Department of chemotherapy ( Site 0803), Saint Petersburg, Leningradskaya Oblast', Russia · ACTIVE_NOT_RECRUITING
• Fed State Budgetary Inst N.N. Blokhin Med Center of Oncology MHRF ( Site 0801), Moscow, Moscow, Russia · ACTIVE_NOT_RECRUITING
• Endocrinology Research Center of Rosmedtechnologies-Surgery ( Site 0809), Moscow, Moscow, Russia · COMPLETED
• National Cancer Centre Singapore ( Site 1700), Singapore, Central Singapore, Singapore · RECRUITING
• Seoul National University Hospital ( Site 2001), Jongno-gu, Seoul, South Korea · RECRUITING
• Asan Medical Center ( Site 2000), Songpa-gu, Seoul, South Korea · RECRUITING
• MD Anderson Cancer Center-Oncology ( Site 1102), Madrid, Madrid, Comunidad de, Spain · COMPLETED
• Hospital Universitario 12 de Octubre-Medical Oncology ( Site 1103), Madrid, Madrid, Comunidad de, Spain · RECRUITING
• Hospital Universitario Central de Asturias-Medical Oncology ( Site 1101), Oviedo, Principality of Asturias, Spain · RECRUITING
• Hospital Universitari Vall d'Hebron ( Site 1100), Barcelona, Spain · RECRUITING
• Skanes University Hospital Lund ( Site 1200), Lund, Skåne County, Sweden · RECRUITING
• Karolinska Universitetssjukhuset Solna ( Site 1202), Stockholm, Stockholm County, Sweden · COMPLETED
• Akademiska sjukhuset-Blod- och tumörsjukdomar ( Site 1201), Uppsala, Uppsala County, Sweden · RECRUITING
• Sahlgrenska Universitetssjukhuset-Department of Oncology CTU Clinical Trial Unit ( Site 1204), Gothenburg, Västra Götaland County, Sweden · RECRUITING
• Baskent University Adana Training Hospital ( Site 0906), Yüreğir, Adana, Turkey (Türkiye) · RECRUITING
• Ege University Medicine of Faculty ( Site 0900), Bornova, İzmir, Turkey (Türkiye) · RECRUITING
• Hacettepe Universitesi-oncology hospital ( Site 0901), Ankara, Turkey (Türkiye) · RECRUITING
• Ankara Bilkent Şehir Hastanesi. ( Site 0904), Ankara, Turkey (Türkiye) · RECRUITING
• Istanbul Universitesi Cerrahpasa-Medical Oncology ( Site 0902), Istanbul, Turkey (Türkiye) · COMPLETED
• Addenbrooke's Hospital ( Site 1309), Cambridge, Cambridgeshire, United Kingdom · RECRUITING
• Royal Free Hospital ( Site 1302), London, England, United Kingdom · ACTIVE_NOT_RECRUITING
• The Beatson West of Scotland Cancer Centre ( Site 1308), Glasgow, Glasgow City, United Kingdom · RECRUITING
• Hammersmith Hospital-Medical Oncology ( Site 1304), London, London, City of, United Kingdom · RECRUITING