Clinical Trials /

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

NCT03401788

Description:

This study is designed to investigate PT2977 as a treatment for VHL disease associated RCC.

Related Conditions:
  • Renal Cell Carcinoma
  • Von Hippel-Lindau Syndrome
Recruiting Status:

Active, not recruiting

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: A Phase 2 Study of PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma
  • Official Title: An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma

Clinical Trial IDs

  • ORG STUDY ID: PT2977-202
  • NCT ID: NCT03401788

Conditions

  • VHL - Von Hippel-Lindau Syndrome
  • VHL
  • VHL Gene Mutation
  • VHL Syndrome
  • VHL Gene Inactivation
  • VHL-Associated Renal Cell Carcinoma
  • VHL-Associated Clear Cell Renal Cell Carcinoma

Interventions

DrugSynonymsArms
PT2977Open Label PT2977

Purpose

This study is designed to investigate PT2977 as a treatment for VHL disease associated RCC.

Detailed Description

      This open-label Phase 2 study will evaluate the efficacy and safety of PT2977 in patients
      with VHL disease who have at least 1 measurable RCC tumor. PT2977 will be administered orally
      and treatment will be continuous. Patients 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.
    

Trial Arms

NameTypeDescriptionInterventions
Open Label PT2977ExperimentalPT2977 is a small molecule inhibitor of HIF-2α, which impairs hypoxic and pseudo-hypoxia signaling in cancer cells.
  • PT2977

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). Patients may have VHL disease-associated tumors in other organ systems

        Exclusion Criteria:

          -  Has received prior treatment with PT2977 or another HIF-2α inhibitor

          -  Has had any systemic anti-cancer therapy (includes anti-VEGF therapy or any systemic
             investigational anti-cancer agent)
      
Maximum Eligible Age:N/A
Minimum Eligible Age:18 Years
Eligible Gender:All
Healthy Volunteers:No

Primary Outcome Measures

Measure:Overall response rate (ORR)
Time Frame:4-5 Years
Safety Issue:
Description:ORR per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST)

Secondary Outcome Measures

Measure:Duration of response (DOR)
Time Frame:4-5 Years
Safety Issue:
Description:
Measure:Time to response (TTR)
Time Frame:4-5 Years
Safety Issue:
Description:
Measure:Progression-free survival (PFS)
Time Frame:4-5 Years
Safety Issue:
Description:
Measure:Time to Surgery (TTS)
Time Frame:4-5 Years
Safety Issue:
Description:

Details

Phase:Phase 2
Primary Purpose:Interventional
Overall Status:Recruiting
Lead Sponsor:Peloton Therapeutics, Inc.

Last Updated

March 20, 2018