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

Primary Purpose

VHL Gene Mutation, VHL, VHL Syndrome

Status
Active
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
PT2385 Tablets
Sponsored by
Peloton Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for VHL Gene Mutation

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • National Institutes of Health Clinical Center

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

PT2385 Tablets

Arm Description

Twenty-five patients will be enrolled in each stage of a two-stage design

Outcomes

Primary Outcome Measures

Overall Response Rate (ORR)
Overall response rate (ORR) in VHL disease-associated ccRCC tumors

Secondary Outcome Measures

Progression Free Survival (PFS)
Progression free survival (PFS) in VHL disease-associated ccRCC tumors
Duration of Response (DOR)
Duration of response (DOR) in VHL disease-associated ccRCC tumors
Time to Response (TTR)
Time to response (TTR) in VHL disease-associated ccRCC tumors
Number of Participants who Experience One or More Adverse Events (AEs)
An AE is defined as any untoward medical occurrence in a patient regardless of its causal relationship to study treatment. An AE can be any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug, whether or not it is considered to be study drug related.
Number of Participants who Discontinue Study Intervention Due to an AE.
An AE is defined as any untoward medical occurrence in a patient regardless of its causal relationship to study treatment. An AE can be any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug, whether or not it is considered to be study drug related.

Full Information

First Posted
March 28, 2017
Last Updated
August 10, 2022
Sponsor
Peloton Therapeutics, Inc.
Collaborators
National Institutes of Health (NIH)
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1. Study Identification

Unique Protocol Identification Number
NCT03108066
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
Study Type
Interventional

2. Study Status

Record Verification Date
August 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
April 24, 2017 (Actual)
Primary Completion Date
November 30, 2023 (Anticipated)
Study Completion Date
November 30, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Peloton Therapeutics, Inc.
Collaborators
National Institutes of Health (NIH)

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Single Group Assignment
Model Description
This is an open-label Phase 2 study that will be conducted with a 2-stage design.
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)

8. Arms, Groups, and Interventions

Arm Title
PT2385 Tablets
Arm Type
Experimental
Arm Description
Twenty-five patients will be enrolled in each stage of a two-stage design
Intervention Type
Drug
Intervention Name(s)
PT2385 Tablets
Other Intervention Name(s)
PT2385, PT-2385, HIF-2a, MK-3795
Intervention Description
PT2385 inhibits HIF-2α and is a novel approach to treatment of VHL disease-associated ccRCC.
Primary Outcome Measure Information:
Title
Overall Response Rate (ORR)
Description
Overall response rate (ORR) in VHL disease-associated ccRCC tumors
Time Frame
4-5 years
Secondary Outcome Measure Information:
Title
Progression Free Survival (PFS)
Description
Progression free survival (PFS) in VHL disease-associated ccRCC tumors
Time Frame
4-5 years
Title
Duration of Response (DOR)
Description
Duration of response (DOR) in VHL disease-associated ccRCC tumors
Time Frame
4-5 years
Title
Time to Response (TTR)
Description
Time to response (TTR) in VHL disease-associated ccRCC tumors
Time Frame
4-5 years
Title
Number of Participants who Experience One or More Adverse Events (AEs)
Description
An AE is defined as any untoward medical occurrence in a patient regardless of its causal relationship to study treatment. An AE can be any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug, whether or not it is considered to be study drug related.
Time Frame
Up to approximately 5 years
Title
Number of Participants who Discontinue Study Intervention Due to an AE.
Description
An AE is defined as any untoward medical occurrence in a patient regardless of its causal relationship to study treatment. An AE can be any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug, whether or not it is considered to be study drug related.
Time Frame
Up to approximately 5 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Merck Sharp & Dohme LLC
Official's Role
Study Director
Facility Information:
Facility Name
National Institutes of Health Clinical Center
City
Bethesda
State/Province
Maryland
ZIP/Postal Code
20892
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
IPD Sharing URL
http://engagezone.msd.com/ds_documentation.php
Citations:
PubMed Identifier
34783716
Citation
Larcher A, Rowe I, Belladelli F, Fallara G, Raggi D, Necchi A, Montorsi F, Capitanio U, Salonia A; OSR VHL Program. Von Hippel-Lindau disease-associated renal cell carcinoma: a call to action. Curr Opin Urol. 2022 Jan 1;32(1):31-39. doi: 10.1097/MOU.0000000000000950.
Results Reference
derived
Links:
URL
http://merckoncologyclinicaltrials.com
Description
Merck Oncology Clinical Trials Information

Learn more about this trial

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

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