Testing Belzutifan for pheochromocytoma/paraganglioma
Official title: 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)
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
Interventions
Belzutifan
Belzutifan, 120 mg, oral, once daily (QD) until progressive disease or discontinuation.
Canadian Sites (2)
Arthur J.E. Child Comprehensive Cancer Centre ( Site 0203)
Calgary, Alberta, Canada
Princess Margaret Cancer Centre ( Site 0202)
Toronto, Ontario, Canada
Eligibility Criteria
See who this study is looking for19 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +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 CohortCohortA group of participants sharing a characteristic, followed together.Read more → 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 criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → 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
Exclusion
- −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
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 2Phase 2A middle-stage study looking at what a treatment does and watching for side effects.Read more → — a middle-stage study in a few hundred people at most, looking at what the treatment does and watching for side effectsSide effectAn unwanted effect thought to be caused by the treatment itself.Read more →.
What is being given or done in this study: Belzutifan.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
- armsInterventionsModule.interventions[].name
How the study is run
There is only one group in this study, so there is no assignment to different treatments.
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
There is one group in this study.
From the trial registry
Built from this field:
- armsInterventionsModule.armGroups[].type
Who the study is looking for
The study lists a minimum age of 12 years, with no upper limit given.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 355 people.
The study is currently expected to finish around December 2029.
The main measurement is taken over: Up to approximately 5.5 years.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Objective Response Rate (ORR) as Assessed by Blinded Independent Central Review (BICR) — measured over Up to approximately 5.5 years.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT04924075 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About BelzutifanDrug
Belzutifan, 120 mg, oral, once daily (QD) until progressive disease or discontinuation.
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: a minimum age of 12 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
This study does not list a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
This study is open-labelOpen-labelEveryone knows which treatment is being given — nothing is hidden.Read more →: everyone knows which treatment is being given, including you and the study team.
There is only one group in this study, so there is no assignment to different treatments.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists a minimum age of 12 years, with no upper limit given.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: Up to approximately 5.5 years.
The study as a whole is currently expected to finish around 2029-12-27.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 355 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 4 locations, including: Calgary, Alberta, Canada; Toronto, Ontario, Canada; Ann Arbor, Michigan, United States; New York, New York, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
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).
Other Sites (2)
University of Michigan ( Site 0126)
Ann Arbor, Michigan, United States
Icahn School of Medicine at Mount Sinai ( Site 0123)
New York, New York, United States
Think this trial might be right for you?
Complete a quick intake form and we will match you with this and other relevant trials based on your medical profile.
See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.