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. |
|
|
Placebo Comparator Phase 2: Placebo |
Participants will receive placebo matched to VX-147. |
|
|
Experimental Phase 3: VX-147 |
Participants will receive VX-147 with the dose to be based on the outcome of Phase 2. |
|
|
Placebo Comparator Phase 3: Placebo |
Participants will receive placebo matched to VX-147. |
|
Recruiting Locations
Vanderbilt University Medical Center, Nephrology Clinical Trials Center
Nashville, Tennessee 37232
Nashville, Tennessee 37232
More Details
- Status
- Recruiting
- Sponsor
- Vertex Pharmaceuticals Incorporated