Clinical Trials /

PT2385 for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma

NCT03108066

Description:

The primary objective of this study is to assess the overall response rate (ORR) of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC) tumors in VHL patients treated with PT2385.

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

Active, not recruiting

Phase:

Phase 2

Trial Eligibility

Document

Title

  • Brief Title: PT2385 for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma
  • Official Title: An Open Label Phase 2 Study to Evaluate PT2385 for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell Carcinoma

Clinical Trial IDs

  • ORG STUDY ID: PT2385-202
  • NCT ID: NCT03108066

Conditions

  • VHL Gene Mutation
  • VHL
  • VHL Syndrome
  • VHL Gene Inactivation
  • Von Hippel
  • Von Hippel-Lindau Disease
  • Von Hippel's Disease
  • Von Hippel-Lindau Syndrome, Modifiers of
  • Clear Cell Renal Cell Carcinoma
  • Clear Cell RCC
  • ccRCC

Interventions

DrugSynonymsArms
PT2385 TabletsPT2385, PT-2385, HIF-2aPT2385 Tablets

Purpose

The primary objective of this study is to assess the overall response rate (ORR) of von Hippel-Lindau (VHL) disease-associated clear cell renal cell carcinoma (ccRCC) tumors in VHL patients treated with PT2385.

Detailed Description

      This open-label Phase 2 study will evaluate the efficacy, safety, PK, and PD of PT2385 in
      patients with VHL disease who have at least 1 measurable VHL disease-associated ccRCC tumor
      (as defined by RECIST 1.1). PT2385 will be administered orally and treatment will be
      continuous. Changes in VHL disease-associated non-ccRCC tumors will also be evaluated.
    

Trial Arms

NameTypeDescriptionInterventions
PT2385 TabletsExperimentalTwenty-five patients will be enrolled in each stage of a two-stage design
  • PT2385 Tablets

Eligibility Criteria

        Inclusion Criteria:

          -  Has at least 1 measurable ccRCC tumor and no solid ccRCC tumor greater than 3.0 cm,
             based on radiologic diagnosis (histologic diagnosis not required); may have VHL
             disease-associated lesions in other organ systems

          -  Has a diagnosis of von Hippel Lindau disease, based on a germline VHL alteration

        Exclusion Criteria:

          -  Has had prior radiotherapy or systemic anti cancer therapy for ccRCC (includes
             anti-VEGF therapy or any systemic investigational anti cancer agent)

          -  Has a prior or concomitant non-VHL disease-associated invasive malignancy with the
             exception of adequately treated basal or squamous cell carcinoma of the skin,
             cervical carcinoma in situ or any other malignancy from which the patient has
             remained disease free for more than 2 years

          -  Has any history of metastatic disease

          -  Has had radiotherapy to any non-ccRCC site within 4 weeks prior to entering the study
             or has not recovered from adverse events (AE)

          -  Has had any surgical procedure for VHL disease or any major surgical procedure
             completed within 4 weeks prior to entering the study or has any surgical lesions from
             recent major surgical procedures that are not well healed
      
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:Overall response rate (ORR) in VHL disease-associated ccRCC tumors

Secondary Outcome Measures

Measure:Progression Free Survival (PFS)
Time Frame:4-5 years
Safety Issue:
Description:Progression free survival (PFS) in VHL disease-associated ccRCC tumors
Measure:Duration of Response (DOR)
Time Frame:4-5 years
Safety Issue:
Description:Duration of response (DOR) in VHL disease-associated ccRCC tumors
Measure:Time to Response (TTR)
Time Frame:4-5 years
Safety Issue:
Description:Time to response (TTR) in VHL disease-associated ccRCC tumors

Details

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

Last Updated

April 25, 2017