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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

Eligibility

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

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.

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 investigator. Other protocol defined Inclusion/Exclusion criteria will 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
Part A (Phase 2/3): VX-147 		Participants will be randomized to receive different doses of VX-147. Participants will receive the selected dose of VX-147 for atleast 96 weeks.
  • Drug: VX-147
    Tablets for oral administration.
Placebo Comparator
Part A (Phase 2/3): Placebo 		Participants will receive placebo matched to VX-147.
  • Drug: Placebo
    Tablets for oral administration.
Experimental
Part B (Phase 3): VX-147 		Participants who complete Part A will receive VX-147 for an additional 96 weeks.
  • Drug: VX-147
    Tablets for oral administration.

Recruiting Locations

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.