A Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease
Vertex Pharmaceuticals Incorporated
Summary
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.
Eligibility
- Age range
- 10–65 years
- Sex
- All
- Healthy volunteers
- No
Key Inclusion Criteria: Part A: * APOL1 genotype of G1/G1, G2/G2, or G1/G2 * Proteinuric kidney disease Part B: \- Completion of Treatment Period in Part A and no permanent discontinuation of study drug. Key Exclusion Criteria: Part A: * 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 Part B: * ESKD (End Stage Kidney Disease) as defined in the protocol. * Any lab abnormality that may pose a safety risk to the participant, as judged by the investi…
Interventions
- DrugVX-147
Tablets for oral administration.
- DrugPlacebo
Tablets for oral administration.
Locations (318)
- Alabama Kidney ResearchAlabaster, Alabama
- EmVenio Research - Mobile Unit - BirminghamBirmingham, Alabama
- Cardiology, P.C.Birmingham, Alabama
- Children's Hospital of AlabamaBirmingham, Alabama
- The Kirklin Clinic - NephrologyBirmingham, Alabama
- Nephrology AssociatesFairhope, Alabama