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
Arm | Description | Assigned 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. |
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More Details
- Status
- Completed
- Sponsor
- Vertex Pharmaceuticals Incorporated