Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis

Purpose

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in participants with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).

Condition

  • Glomerulosclerosis, Focal Segmental

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • APOL1 genotype of G1/G1, G2/G2, or G1/G2 - FSGS diagnosed by kidney biopsy

Exclusion Criteria

  • Evidence of non-APOL1-mediated FSGS - Participants with known sickle cell disease - Solid organ or Bone marrow transplant Other protocol defined Inclusion/Exclusion criteria may apply.

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
N/A
Intervention Model
Single Group Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
VX-147
All participants received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.
  • Drug: VX-147
    Tablets for oral administration.
    Other names:
    • IXP

More Details

Status
Completed
Sponsor
Vertex Pharmaceuticals Incorporated

Study Contact