Description:
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.
Title
- 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
Clinical Trial IDs
- ORG STUDY ID:
3795-003
- SECONDARY ID:
PT2385-202
- NCT ID:
NCT03108066
Conditions
- 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
Interventions
Drug | Synonyms | Arms |
---|
PT2385 Tablets | PT2385, PT-2385, HIF-2a, MK-3795 | PT2385 Tablets |
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.
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.
Trial Arms
Name | Type | Description | Interventions |
---|
PT2385 Tablets | Experimental | Twenty-five patients will be enrolled in each stage of a two-stage design | |
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
Maximum Eligible Age: | N/A |
Minimum Eligible Age: | 18 Years |
Eligible Gender: | All |
Healthy Volunteers: | No |
Primary Outcome Measures
Measure: | Overall Response Rate (ORR) |
Time Frame: | 4-5 years |
Safety Issue: | |
Description: | Overall response rate (ORR) in VHL disease-associated ccRCC tumors |
Secondary Outcome Measures
Measure: | Progression Free Survival (PFS) |
Time Frame: | 4-5 years |
Safety Issue: | |
Description: | Progression free survival (PFS) in VHL disease-associated ccRCC tumors |
Measure: | Duration of Response (DOR) |
Time Frame: | 4-5 years |
Safety Issue: | |
Description: | Duration of response (DOR) in VHL disease-associated ccRCC tumors |
Measure: | Time to Response (TTR) |
Time Frame: | 4-5 years |
Safety Issue: | |
Description: | Time to response (TTR) in VHL disease-associated ccRCC tumors |
Details
Phase: | Phase 2 |
Primary Purpose: | Interventional |
Overall Status: | Active, not recruiting |
Lead Sponsor: | Peloton Therapeutics, Inc. |
Last Updated
April 13, 2021