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)
Florida Premier Research Institute
🇺🇸Winter Park, Florida, United States
Central Georgia Kidney Specialists PC
🇺🇸Macon, Georgia, United States
Temple University Hospital
🇺🇸Philadelphia, Pennsylvania, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
California Institute of Renal Research
🇺🇸San Diego, California, United States
Kidney and Hypertension Specialists of Miami
🇺🇸Miami, Florida, United States
Boston Medical Center
🇺🇸Boston, Massachusetts, United States
Privia Medical Group
🇺🇸Houston, Texas, United States
Beth Israel Deaconess Medical Center
🇺🇸Boston, Massachusetts, United States
Nevada Kidney Disease and Hypertension Centers
🇺🇸Las Vegas, Nevada, United States
Prolato Clinical Research Center
🇺🇸Houston, Texas, United States
University of Alabama at Birmingham - The Kirklin Clinic
🇺🇸Birmingham, Alabama, United States
Durham Nephrology Associates, PA
🇺🇸Durham, North Carolina, United States
Duke University School of Medicine - Duke Molecular Physiology Institute
🇺🇸Durham, North Carolina, United States
Vanderbilt University VU
🇺🇸Nashville, Tennessee, 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
Morehouse School of Medicine, Grady Memorial Hospital
🇺🇸Atlanta, Georgia, United States
Georgia Nehphrology
🇺🇸Lawrenceville, Georgia, United States
Ochsner Medical Center - New Orleans
🇺🇸New Orleans, Louisiana, United States
St Louis Kidney Care
🇺🇸Saint Louis, Missouri, United States
Paragon Health, PC d/b/a Nephrology Center, PC
🇺🇸Kalamazoo, Michigan, United States
Nephrology and Hypertension Associates, LTD
🇺🇸Tupelo, Mississippi, United States
Renal and Transplant Associates of New England, PC
🇺🇸Springfield, Massachusetts, United States
SUNY Downstate
🇺🇸Brooklyn, New York, United States
Urban Family Practice
🇺🇸Buffalo, New York, United States
UNC Kidney Center Division of Nephrology & Hypertension
🇺🇸Chapel Hill, North Carolina, United States
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States
Tryon Medical Partners
🇺🇸Charlotte, North Carolina, United States
Hopital Henri Mondor
🇫🇷Créteil, France
Children's Hospital of Pittsburgh of UPMC
🇺🇸Pittsburgh, Pennsylvania, United States
South Carolina Nephrology and Hypertension Center, Inc.
🇺🇸Orangeburg, South Carolina, United States
Medical University of South Carolina
🇺🇸Charleston, South Carolina, United States
Hôpital Bicêtre AP-HP
🇫🇷Le Kremlin-Bicêtre, France
Bichat Hospital
🇫🇷Paris, France
Service de Nephrologie - Hopital Universitaire Necker
🇫🇷Paris, France
Hopitaux Universitaires Est Parisien - Hopital Tenon
🇫🇷Paris, France
GCM Medical Group, PSC
🇵🇷San Juan, Puerto Rico
King's College Hospital NHS Foundation Trust - Guthrie Clinic
🇬🇧Southwark, United Kingdom
Université Paris-Descartes / Hôpital Européen Georges Pompidou
🇫🇷Paris, France
Barts Health NHS Trust
🇬🇧London, United Kingdom
University Hospitals of Leicester NHS Trust - Leicester General Hospital
🇬🇧Leicester, United Kingdom
The Medicines Evaluation Unit
🇬🇧Manchester, United Kingdom
Renal Associates of Baton Rouge
🇺🇸Baton Rouge, Louisiana, United States