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)

Peloton Therapeutics

Status and phase: Active, not recruiting; Phase 2

Conditions: VHL-Associated Renal Cell Carcinoma; VHL-Associated Clear Cell Renal Cell Carcinoma; VHL Gene Inactivation; VHL Syndrome; VHL - Von Hippel-Lindau Syndrome; VHL Gene Mutation

Treatments: Drug: Belzutifan

Study type: Interventional

Funder types: Industry

Identifiers

NCT03401788

2023-509119-99 (Registry Identifier)

2018-000125-30 (EudraCT Number)

6482-004

MK-6482-004 (Other Identifier)

PT2977-202 (Other Identifier)

Details and patient eligibility

About

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

Full description

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.

Enrollment: 50 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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50 participants in 1 patient group

Open Label Belzutifan

Experimental group

Description:

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.

Treatment:

Drug: Belzutifan