A Phase II 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

Study Identifier:
MK-6482-015
CT.gov Identifier:
NCT04924075
EudraCT Identifier:
202350485311
EU Trial (CTIS) Number:
2023-504853-11-00
Study Contact Information:
(210) 593-5651 or [email protected]
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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 Gastrointestinal Stromal Tumor (wt GIST), or Advanced Solid Tumors With hypoxia inducible factor-2 alpha (HIF-2α) related genetic alterations.

To evaluate the efficacy and safety of Belzutifan monotherapy in participants with advanced pheochromocytoma/paraganglioma (PPGL) or pancreatic neuroendocrine tumor (pNET).

To evaluate belzutifan in participants (pts) with advanced panNETs.

To evaluate the HIF-2α inhibitor, belzutifan, in advanced PPGL.

Medical Condition
The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.
Renal
Solid Tumor
Gastrointestinal stromal tumor
Neuroendocrine
Phase
The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
Phase II
Sex
Female & Male
Age
12+ years
Study Drug
Drug: Arms:
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Drug: Experimental drug
Drug: Patients received oral belzutifan 120 mg once daily until PD or unacceptable toxicity.
Study Status
Indicates the current recruitment status or the expanded access status
Recruiting

Requirements information
Inclusion 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
Exclusion criteria
  • 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

Clinical Study Information for Healthcare Providers

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Study Locations

Location
Investigator
Status
Condition(s) Treated at Site
Location
START Lisbon
Lisbon, Portugal, 1649-035
Investigator
Andre Mansinho
Status
Recruiting
Condition(s) Treated at Site
Renal
Solid Tumor
Gastrointestinal stromal tumor
Neuroendocrine