A Phase 2 Study of PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma

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Brief Title

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)

Official Title

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

Brief Summary

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

Detailed 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. Changes in VHL disease-associated non-RCC tumors
      will also be evaluated.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Objective Response Rate (ORR) in VHL Disease-Associated RCC Tumors

Secondary Outcome

 Duration of Response (DOR) in VHL Disease-Associated RCC Tumors

Condition

VHL - Von Hippel-Lindau Syndrome

Intervention

Belzutifan

Study Arms / Comparison Groups

 Open Label Belzutifan
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.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

50

Start Date

May 2, 2018

Completion Date

March 29, 2022

Primary Completion Date

March 29, 2022

Eligibility Criteria

        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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

Denmark

Location Countries

Denmark

Administrative Informations


NCT ID

NCT03401788

Organization ID

6482-004

Secondary IDs

PT2977-202

Responsible Party

Sponsor

Study Sponsor

Peloton Therapeutics, Inc.


Study Sponsor

, , 


Verification Date

October 2020