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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 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

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.