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

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ClinicalTrials.gov Identifier: NCT03401788
Recruitment Status : Recruiting
First Posted : January 17, 2018
Last Update Posted : November 14, 2018
Sponsor:
Information provided by (Responsible Party):
Peloton Therapeutics, Inc.

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

Condition or disease Intervention/treatment Phase
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 Drug: PT2977 Phase 2

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.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Phase 2 Open Label
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel Lindau Disease-Associated Renal Cell Carcinoma
Actual Study Start Date : March 20, 2018
Estimated Primary Completion Date : March 1, 2020
Estimated Study Completion Date : March 1, 2023


Arm Intervention/treatment
Experimental: Open Label PT2977
PT2977 is a small molecule inhibitor of HIF-2α, which impairs hypoxic and pseudo-hypoxia signaling in cancer cells.
Drug: PT2977
HIF-2α Inhibitor




Primary Outcome Measures :
  1. Overall response rate (ORR) [ Time Frame: 4-5 Years ]
    ORR per Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST)


Secondary Outcome Measures :
  1. Duration of response (DOR) [ Time Frame: 4-5 Years ]
  2. Time to response (TTR) [ Time Frame: 4-5 Years ]
  3. Progression-free survival (PFS) [ Time Frame: 4-5 Years ]
  4. Time to Surgery (TTS) [ Time Frame: 4-5 Years ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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)
  • Has an immediate need for surgical intervention for tumor treatment
  • Has evidence of metastatic disease on screening imaging

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 ClinicalTrials.gov identifier (NCT number): NCT03401788


Contacts
Contact: Richard Kelley, MBA 972-629-4088 richard.kelley@pelotontx.com

Locations
United States, Colorado
University of Colorado Cancer Center Not yet recruiting
Aurora, Colorado, United States, 80045
United States, Connecticut
Yale Cancer Center Not yet recruiting
New Haven, Connecticut, United States, 06519
United States, Maryland
National Institutes of Health Clinical Center Recruiting
Bethesda, Maryland, United States, 20892
Contact: Laura Wisch    301-480-6194    laura.wisch@nih.gov   
Principal Investigator: Ramaprasad Srinivasan, MD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
United States, Michigan
University of Michigan Recruiting
Ann Arbor, Michigan, United States, 48109
Contact: Cancer Answer Line    800-865-1125    canceranswerline@med.umich.edu   
United States, New York
Memorial Sloan Kettering Cancer Center Not yet recruiting
New York, New York, United States, 10065
United States, North Carolina
Wake Forest Not yet recruiting
Winston-Salem, North Carolina, United States, 27103
United States, Pennsylvania
University of Pennsylvania Medical Center Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Mary Walicki    215-662-3510    Mary.walicki@uphs.upenn.edu   
University of Pittsburgh Medical Center Recruiting
Pittsburgh, Pennsylvania, United States, 15232
Contact: Dawn McBride    412-623-2764    mcbridedl@upmc.edu   
United States, Tennessee
Vanderbilt Medical Center Recruiting
Nashville, Tennessee, United States, 37232
Contact: Eligibility Office    800-811-8480      
United States, Texas
UT Southwestern Medical Center Recruiting
Dallas, Texas, United States, 75239
Contact: Allison Beaver    214-645-8787    Allison.Beaver@utsouthwestern.edu   
MD Anderson Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Christine Robichaux    713-794-4077    crobichaux@mdanderson.org   
United States, Utah
Huntsman Cancer Institute Recruiting
Salt Lake City, Utah, United States, 84112
Contact: Samantha Greenberg    801-213-5774      
Contact       vhlclinic@hci.utah.edu   
Denmark
Aarhus University Hospital Not yet recruiting
Aarhus, Denmark
France
Hospital Georges Pompidou Not yet recruiting
Paris, France
Netherlands
University Medical Center Utrecht Not yet recruiting
Utrecht, Netherlands
United Kingdom
Cambridge University Hospitals Not yet recruiting
Cambridge, United Kingdom
Guy's and St. Thomas' Hospitals Not yet recruiting
London, United Kingdom
Christie NHS Foundation Trust Not yet recruiting
Manchester, United Kingdom
Sponsors and Collaborators
Peloton Therapeutics, Inc.

Responsible Party: Peloton Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT03401788     History of Changes
Other Study ID Numbers: PT2977-202
First Posted: January 17, 2018    Key Record Dates
Last Update Posted: November 14, 2018
Last Verified: October 2018
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