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]
Recruiting

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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
  • Cohort A3
  • : Patients aged 12 years or older at the time of informed consent
  • , diagnosed with advanced pheochromocytoma/paraganglioma
  • , and expected to survive for 3 months or more.
  • Cohort B1 (enrollment for Cohort B1 has been completed)
  • : Patients confirmed to have VHL disease by germline testing at the clinical trial site, or patients with a clinical diagnosis of VHL disease,
  • aged 18 years or older at the time of informed consent,
  • and expected to survive for 3 months or more.
Exclusion criteria
  • ・Patients with diseases that make oral administration difficult or that may affect the absorption of MK-6482.
  • ・Patients with a history of secondary cancer. However, if curative treatment has been completed and no signs of malignancy have been observed for two years, they may be included, with the following exceptions:
  • ・Patients who meet any of the following conditions:
  • ○ Resting pulse oximeter reading is less than 92%, or
  • ○ Requires intermittent oxygen supplementation, or
  • ○ Requires chronic oxygen supplementation.
  • ・Patients who have clinically significant heart disease (unstable angina, acute myocardial infarction, coronary artery bypass surgery, percutaneous coronary intervention, etc.) or congestive heart failure of New York Heart Association functional classification Class III or IV within six months prior to the first day of administration
  • of the investigational drug. ・Patients who have received prior treatment with chemotherapy, targeted therapy, biological agents, or other investigational drugs (excluding somatostatin analogs for pNET patients) within four weeks prior to the first administration of the investigational drug.

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