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Treatment of advanced tumors with belzutifan

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

Phase 2 Interventional Merck Sharp & Dohme LLC · NCT04924075

This study is testing a new medication called belzutifan to see if it can help people with advanced tumors related to specific conditions feel better and respond to treatment.

Quick facts

PhasePhase 2
Study typeInterventional
Enrollment322 (estimated)
Ages12 Years and up
SexAll
SponsorMerck Sharp & Dohme LLC Industry-sponsored
Drugs / interventionschemotherapy
Locations84 sites (Los Angeles, California and 83 other locations)
Trial IDNCT04924075 on ClinicalTrials.gov

What this trial studies

This study evaluates the efficacy and safety of belzutifan, a medication targeting hypoxia inducible factor-2 alpha (HIF-2α), in patients with advanced pheochromocytoma/paraganglioma, pancreatic neuroendocrine tumors, von Hippel-Lindau disease-associated tumors, advanced gastrointestinal stromal tumors, or solid tumors with HIF-2α related genetic alterations. The primary objective is to assess the objective response rate (ORR) of belzutifan using established response evaluation criteria. Participants will be monitored for safety and treatment response through blinded independent central review.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 12 and older with advanced pheochromocytoma/paraganglioma, pancreatic neuroendocrine tumors, or other specified tumors related to HIF-2α.

Not a fit: Patients with a history of second malignancies or those unable to take oral medications may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could provide a new therapeutic option for patients with advanced tumors associated with HIF-2α alterations.

How similar studies have performed: Other studies targeting HIF-2α have shown promise, indicating potential for success with this approach.

Eligibility criteria

Show full inclusion / exclusion 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 BI: 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

Where this trial is running

Los Angeles, California and 83 other locations

+34 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Pheochromocytoma/ParagangliomaPancreatic Neuroendocrine TumorVon Hippel-Lindau DiseaseAdvanced Gastrointestinal Stromal TumorHIF-2α Mutated CancersHIF-2αPheochromocytoma/paragangliomaPancreatic NET
Last reviewed 2026-06-10 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.