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

ArmDescriptionAssigned 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

University of Kansas Medical Center
Kansas City, Kansas 66160

More Details

Status
Recruiting
Sponsor
Vertex Pharmaceuticals Incorporated

Study Contact

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