Vanderbilt Clinical Trials

Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1- Mediated Proteinuric Kidney Disease Mediated Proteinuric Kidney Disease

Purpose

The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of VX-147 in adult and pediatric participants with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.

Condition

Proteinuric Kidney Disease

Eligibility

Eligible Ages: Between 10 Years and 65 Years
Eligible Genders: All
Accepts Healthy Volunteers: No

Inclusion Criteria

APOL1 genotype of G1/G1, G2/G2, or G1/G2 - Proteinuric kidney disease

Exclusion Criteria

Solid organ or bone marrow transplant - Uncontrolled hypertension - History of diabetes mellitus - Known underlying cause of kidney disease including but not limited to sickle cell disease Other protocol defined Inclusion/Exclusion criteria apply.

Study Design

Phase: Phase 2/Phase 3
Study Type: Interventional
Allocation: Randomized
Intervention Model: Sequential Assignment
Primary Purpose: Treatment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Arm Groups

Arm	Description	Assigned Intervention
Experimental Phase 2: VX-147	Participants will be randomized to receive different dose levels of VX-147.	Drug: VX-147 Tablets for oral administration.
Placebo Comparator Phase 2: Placebo	Participants will receive placebo matched to VX-147.	Drug: Placebo Tablets for oral administration.
Experimental Phase 3: VX-147	Participants will receive VX-147 with the dose to be based on the outcome of Phase 2.	Drug: VX-147 Tablets for oral administration.
Placebo Comparator Phase 3: Placebo	Participants will receive placebo matched to VX-147.	Drug: Placebo Tablets for oral administration.

Recruiting Locations

Vanderbilt University Medical Center, Nephrology Clinical Trials Center
Nashville, Tennessee 37232

More Details

Status: Recruiting
Sponsor: Vertex Pharmaceuticals Incorporated

Study Contact

Medical Information
617-341-6777
medicalinfo@vrtx.com

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.