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

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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

Brief Summary

      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.
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Overall Response Rate (ORR)

Secondary Outcome

 Progression Free Survival (PFS)

Condition

VHL Gene Mutation

Intervention

PT2385 Tablets

Study Arms / Comparison Groups

 PT2385 Tablets
Description:  Twenty-five patients will be enrolled in each stage of a two-stage design

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

4

Start Date

April 24, 2017

Completion Date

June 15, 2022

Primary Completion Date

June 15, 2022

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
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT03108066

Organization ID

3795-003

Secondary IDs

PT2385-202

Responsible Party

Sponsor

Study Sponsor

Peloton Therapeutics, Inc.

Collaborators

 National Institutes of Health (NIH)

Study Sponsor

, , 


Verification Date

April 2021