Suspended

An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma

What is being tested

Belzutifan

Drug

Who is being recruted

Congenital Abnormalities+8

+ Abnormalities, Multiple

+ Ciliopathies

Over 18 Years

+6 Eligibility Criteria

See all eligibility criteria

How is the trial designed

Treatment Study

Phase 2

Interventional

Study Start: May 2018

See protocol details

Summary

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

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Study start date: May 2, 2018

Actual date on which the first participant was enrolled.

This open-label Phase 2 study will evaluate the efficacy and safety of belzutifan in participants with VHL disease who have at least 1 measurable RCC tumor. Belzutifan will be administered orally and treatment will be continuous. Participants will be evaluated radiologically approximately 12 weeks after initiation of treatment and every 12 weeks thereafter while continuing in the study for a minimum of 3 years and then every 24 weeks or more frequently if clinically indicated. Changes in VHL disease-associated non-RCC tumors will also be evaluated.

Official TitleAn Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma

NCT03401788

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

Last updated: January 28, 2026

Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.

Design Details

50 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.

Conditions

Criteria

Any sex

Biological sex of participants that are eligible to enroll.

Over 18 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

Congenital AbnormalitiesAbnormalities, MultipleCiliopathiesAngiomatosisCardiovascular Diseasesvon Hippel-Lindau DiseaseCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesNervous System DiseasesVascular DiseasesNeurocutaneous SyndromesGenetic Diseases, Inborn

Criteria

2 inclusion criteria required to participate

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

4 exclusion criteria prevent from participating

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

Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.

Treatment Groups

Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group

Treatment Groups

Group I

Experimental

Participants receive 120 mg belzutifan orally once daily. Participants may continue to receive belzutifan in the absence of unacceptable treatment related toxicity or unequivocal disease progression.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 11 locations

Suspended

National Institutes of Health Clinical Center

Bethesda, United StatesOpen National Institutes of Health Clinical Center in Google Maps

Suspended

Massachusetts General Hospital

Boston, United States

Suspended

University of Michigan

Ann Arbor, United States

Suspended

University of Pennsylvania Medical Center

Philadelphia, United States

Suspended11 Study Centers