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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03108066
Recruitment Status : Active, not recruiting
First Posted : April 11, 2017
Last Update Posted : November 15, 2018
National Institutes of Health (NIH)
Information provided by (Responsible Party):
Peloton Therapeutics, Inc.

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.

Condition or disease Intervention/treatment Phase
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 Drug: PT2385 Tablets Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Intervention Model: Single Group Assignment
Intervention Model Description: This is an open-label Phase 2 study that will be conducted with a 2-stage design.
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : May 10, 2017
Estimated Primary Completion Date : April 2022
Estimated Study Completion Date : September 2022

Arm Intervention/treatment
Experimental: PT2385 Tablets
Twenty-five patients will be enrolled in each stage of a two-stage design
Drug: PT2385 Tablets
PT2385 inhibits HIF-2α and is a novel approach to treatment of VHL disease-associated ccRCC.
Other Name: PT2385, PT-2385, HIF-2a

Primary Outcome Measures :
  1. Overall Response Rate (ORR) [ Time Frame: 4-5 years ]
    Overall response rate (ORR) in VHL disease-associated ccRCC tumors

Secondary Outcome Measures :
  1. Progression Free Survival (PFS) [ Time Frame: 4-5 years ]
    Progression free survival (PFS) in VHL disease-associated ccRCC tumors

  2. Duration of Response (DOR) [ Time Frame: 4-5 years ]
    Duration of response (DOR) in VHL disease-associated ccRCC tumors

  3. Time to Response (TTR) [ Time Frame: 4-5 years ]
    Time to response (TTR) in VHL disease-associated ccRCC tumors

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03108066

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United States, Maryland
National Institutes of Health Clinical Center
Bethesda, Maryland, United States, 20892
Sponsors and Collaborators
Peloton Therapeutics, Inc.
National Institutes of Health (NIH)

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Responsible Party: Peloton Therapeutics, Inc. Identifier: NCT03108066     History of Changes
Other Study ID Numbers: PT2385-202
First Posted: April 11, 2017    Key Record Dates
Last Update Posted: November 15, 2018
Last Verified: November 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Carcinoma, Renal Cell
Von Hippel-Lindau Disease
Pathologic Processes
Neoplasms, Glandular and Epithelial
Neoplasms by Histologic Type
Kidney Neoplasms
Urologic Neoplasms
Urogenital Neoplasms
Neoplasms by Site
Kidney Diseases
Urologic Diseases
Neurocutaneous Syndromes
Nervous System Diseases
Vascular Diseases
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn