Health Trials

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)

ConditionsPheochromocytoma/ParagangliomaPancreatic Neuroendocrine TumorVon Hippel-Lindau DiseaseAdvanced Gastrointestinal Stromal TumorHIF-2α Mutated Cancers
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age12+
SponsorMerck Sharp & Dohme LLC

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).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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

Have a diagnosis of VHL disease as determined by a germline test locally and/or clinical diagnosis

Must be ≥18 years of age

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • Belzutifan

    Drug

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

Treatment groups

355 Participants
are divided into 1 treatment group
Group A: BelzutifanExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Objective Response Rate (ORR) as Assessed by Blinded Independent Central Review (BICR)

ORR is the percentage of participants with complete response (CR: disappearance of all target lesions) or partial response (PR: at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of the diameters of target lesions) until progressive disease (PD, at least a 20% increase in the sum of diameters of target lesions, taking as reference the smallest sum on study. The appearance of one or more new lesions is also considered progression) or death due to any cause, whichever occurs first.

Time frame
Up to approximately 5.5 years

Secondary outcomes

1

Duration of Response (DOR) as Assessed by BICR

DOR is the time from first documented evidence of CR or PR until disease progression or death due to any cause, whichever occurs first.

Time frame
Up to approximately 5.5 years
2

Time to Response (TTR) as Assessed by BICR

TTR is defined as the time from first dose of belzutifan to first documented evidence of CR or PR.

Time frame
Up to approximately 5.5 years
3

Disease Control Rate (DCR) as Assessed by BICR

Disease control is a confirmed CR, PR, or stable disease (SD, neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study).

Time frame
Up to approximately 5.5 years
4

Progressive Free Survival (PFS) as Assessed by BICR

PFS is the time from first dose of belzutifan to the first documented PD or death from any cause, whichever occurs first.

Time frame
Up to approximately 5.5 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Merck Sharp & Dohme LLC

Lead sponsor

Source ClinicalTrials.gov
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