A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004)

NCT03401788 · tracked via the Priya Life Science USA tracker

Sponsor

Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

Phase

Phase 2

Started

2018-05-02

Last updated

2026-04-13

Condition(s) studied

VHL - Von Hippel-Lindau SyndromeVHL Gene MutationVHL SyndromeVHL Gene InactivationVHL-Associated Renal Cell CarcinomaVHL-Associated Clear Cell Renal Cell Carcinoma

Investigational drug(s) / intervention(s)

Belzutifan

Belzutifan: 120 mg once daily (three 40 mg oral tablets once daily).

Study summary

This study is designed to investigate belzutifan as a treatment for VHL disease associated RCC.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

Inclusion Criteria: * Has a diagnosis of von Hippel Lindau disease, based on a germline VHL alteration * Has at least 1 measurable solid RCC tumor and no RCC tumor that requires immediate surgical intervention. The diagnosis of RCC can be radiologic (histologic diagnosis not required). Participants may have VHL disease-associated tumors in other organ systems Exclusion Criteria: * Has received prior treatment with belzutifan or another HIF-2α inhibitor * Has had any systemic anti-cancer therapy (includes anti-vascular endothelial growth factor \[VEGF\] therapy or any systemic investigational anti-cancer agent) * Has an immediate need for surgical intervention for tumor treatment * Has evidence of metastatic disease on screening imaging

Primary outcome measure(s)

Objective Response Rate (ORR) in VHL Disease-Associated RCC Tumors — Up to approximately 4 years
ORR is defined as the percentage of participants in the analysis population who have a best confirmed response of Complete Response (CR: Disappearance of all target lesions) or a Partial Response (PR: At least a 30% decrease in the sum of diameters of target lesions) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1). ORR will be assessed by independent review committee (ICR) for the primary analysis.

Trial sites (11)

Facility	City	Region
National Institutes of Health Clinical Center	Bethesda	Maryland
Massachusetts General Hospital	Boston	Massachusetts
University of Michigan	Ann Arbor	Michigan
University of Pennsylvania Medical Center	Philadelphia	Pennsylvania
University of Pittsburgh Medical Center	Pittsburgh	Pennsylvania
Vanderbilt Medical Center	Nashville	Tennessee
MD Anderson Cancer Center	Houston	Texas
Huntsman Cancer Institute	Salt Lake City	Utah
Aarhus University Hospital	Aarhus	Denmark
Hospital Georges Pompidou	Paris	France
Cambridge University Hospital	Cambridge	United Kingdom

More Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) trials in the USA

A Phase 1, Dose-Escalation Trial of PT2385 Tablets In Patients With Advanced Clear Cell Renal Cell Carcinoma (MK-3795-001) Active, not recruiting

A Trial of Belzutifan (PT2977, MK-6482) in Combination With Cabozantinib in Patients With Clear Cell Renal Cell Carcinoma (ccRCC) (MK-6482-003) Active, not recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT03401788 on ClinicalTrials.gov ↗ ← All trials in the USA