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

This study is currently recruiting participants.
See Contacts and Locations
Verified April 2017 by Peloton Therapeutics, Inc.
Sponsor:
Collaborator:
National Institutes of Health (NIH)
Information provided by (Responsible Party):
Peloton Therapeutics, Inc.
ClinicalTrials.gov Identifier:
NCT03108066
First received: March 28, 2017
Last updated: April 25, 2017
Last verified: April 2017
  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.

Condition Intervention 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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
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: No masking
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

Resource links provided by NLM:


Further study details as provided by Peloton Therapeutics, Inc.:

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


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

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

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


Estimated Enrollment: 50
Anticipated Study Start Date: April 2017
Estimated Study Completion Date: September 2022
Estimated Primary Completion Date: April 2022 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

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.
  Eligibility

Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03108066

Contacts
Contact: Richard Kelley 972-629-4081 clinicalstudies@pelotontx.com

Locations
United States, Maryland
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: Martha Ninos, RN    301-435-8897    mninos@mail.nih.gov   
Principal Investigator: Ramaprasad Srinivasan, MD         
Sponsors and Collaborators
Peloton Therapeutics, Inc.
National Institutes of Health (NIH)
  More Information

Responsible Party: Peloton Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03108066     History of Changes
Other Study ID Numbers: PT2385-202
Study First Received: March 28, 2017
Last Updated: April 25, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

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

ClinicalTrials.gov processed this record on July 21, 2017