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

Clear Cell Renal Cell Carcinoma Clinical Trial

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 Details — Status: Completed

Administrative data

• NCT number: NCT03108066
• Other study ID #: 3795-003
• Secondary ID: PT2385-202
• Status: Completed
• Phase: Phase 2
• Start date: April 24, 2017
• Est. completion date: September 27, 2023

Study information

• Verified date: December 2023
• Source: Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: September 27, 2023
• Est. primary completion date: August 30, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Carcinoma
• Carcinoma, Renal Cell
• ccRCC
• Clear Cell RCC
• Clear Cell Renal Cell Carcinoma
• Syndrome
• VHL
• VHL Gene Inactivation
• VHL Gene Mutation
• VHL Syndrome
• Von Hippel
• Von Hippel's Disease
• Von Hippel-Lindau Disease
• Von Hippel-Lindau Syndrome, Modifiers of

Intervention

Drug:
• PT2385 Tablets
PT2385 inhibits HIF-2a and is a novel approach to treatment of VHL disease-associated ccRCC.

Locations

Country	Name	City	State
United States	National Institutes of Health Clinical Center	Bethesda	Maryland

Sponsors (2)

Lead Sponsor	Collaborator
Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)	National Institutes of Health (NIH)

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall Response Rate (ORR)	Overall response rate (ORR) in VHL disease-associated ccRCC tumors	4-5 years
Secondary	Progression Free Survival (PFS)	Progression free survival (PFS) in VHL disease-associated ccRCC tumors	4-5 years
Secondary	Duration of Response (DOR)	Duration of response (DOR) in VHL disease-associated ccRCC tumors	4-5 years
Secondary	Time to Response (TTR)	Time to response (TTR) in VHL disease-associated ccRCC tumors	4-5 years
Secondary	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.	Up to approximately 5 years
Secondary	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.	Up to approximately 5 years

External Links

•   Merck Clinical Trials Information