Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
- Registration Number
- NCT04340362
- Lead Sponsor
- Vertex Pharmaceuticals Incorporated
- Brief Summary
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in participants with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 16
- APOL1 genotype of G1/G1, G2/G2, or G1/G2
- FSGS diagnosed by kidney biopsy
Key
- 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
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description VX-147 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.
- Primary Outcome Measures
Name Time Method Percent Change From Baseline in UPCR From Baseline up to Week 13
- Secondary Outcome Measures
Name Time Method Maximum Observed Concentration (Cmax) of VX-147 Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5 Observed Pre-dose Concentration (Ctrough) of VX-147 Pre-dose on Day 8, 15, Week 3, 5, 9 and 13 Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) From Baseline up to Week 17 Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147 Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5
Trial Locations
- Locations (44)
University of Alabama at Birmingham - The Kirklin Clinic
🇺🇸Birmingham, Alabama, United States
California Institute of Renal Research
🇺🇸San Diego, California, United States
The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension
🇺🇸Washington, District of Columbia, United States
Howard University Hospital
🇺🇸Washington, District of Columbia, United States
Kidney and Hypertension Specialists of Miami
🇺🇸Miami, Florida, United States
Florida Premier Research Institute
🇺🇸Winter Park, Florida, United States
Morehouse School of Medicine, Grady Memorial Hospital
🇺🇸Atlanta, Georgia, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Georgia Nehphrology
🇺🇸Lawrenceville, Georgia, United States
Central Georgia Kidney Specialists PC
🇺🇸Macon, Georgia, United States
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