Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
A Phase 2a, Open-label, Single-arm, 2-Part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis
2 other identifiers
interventional
16
4 countries
44
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_2
Started Jun 2020
Shorter than P25 for phase_2
44 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
April 7, 2020
CompletedFirst Posted
Study publicly available on registry
April 9, 2020
CompletedStudy Start
First participant enrolled
June 8, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
November 11, 2021
CompletedStudy Completion
Last participant's last visit for all outcomes
December 9, 2021
CompletedResults Posted
Study results publicly available
July 10, 2023
CompletedSeptember 24, 2025
September 1, 2025
1.4 years
April 7, 2020
April 28, 2023
September 22, 2025
Conditions
Outcome Measures
Primary Outcomes (1)
Percent Change From Baseline in UPCR
From Baseline up to Week 13
Secondary Outcomes (4)
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
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
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
Study Arms (1)
VX-147
EXPERIMENTALAll 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.
Interventions
Eligibility Criteria
You may qualify if:
- APOL1 genotype of G1/G1, G2/G2, or G1/G2
- FSGS diagnosed by kidney biopsy
You may not qualify if:
- Evidence of non-APOL1-mediated FSGS
- Participants with known sickle cell disease
- Solid organ or Bone marrow transplant
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (44)
University of Alabama at Birmingham - The Kirklin Clinic
Birmingham, Alabama, 35294, United States
California Institute of Renal Research
San Diego, California, 92123, United States
The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension
Washington D.C., District of Columbia, 20037, United States
Howard University Hospital
Washington D.C., District of Columbia, 20060, United States
Kidney and Hypertension Specialists of Miami
Miami, Florida, 33150, United States
Florida Premier Research Institute
Winter Park, Florida, 32789, United States
Morehouse School of Medicine, Grady Memorial Hospital
Atlanta, Georgia, 30310, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
Georgia Nehphrology
Lawrenceville, Georgia, 30046, United States
Central Georgia Kidney Specialists PC
Macon, Georgia, 31201, United States
Renal Associates of Baton Rouge
Baton Rouge, Louisiana, 70808, United States
Ochsner Medical Center - New Orleans
New Orleans, Louisiana, 70121, United States
Boston Medical Center
Boston, Massachusetts, 02118, United States
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215, United States
Renal and Transplant Associates of New England, PC
Springfield, Massachusetts, 01107, United States
Paragon Health, PC d/b/a Nephrology Center, PC
Kalamazoo, Michigan, 49007, United States
Nephrology and Hypertension Associates, LTD
Tupelo, Mississippi, 38801, United States
St Louis Kidney Care
St Louis, Missouri, 63136, United States
Nevada Kidney Disease and Hypertension Centers
Las Vegas, Nevada, 89106, United States
SUNY Downstate
Brooklyn, New York, 11203, United States
Urban Family Practice
Buffalo, New York, 14201, United States
Icahn School of Medicine at Mount Sinai
New York, New York, 10029, United States
UNC Kidney Center Division of Nephrology & Hypertension
Chapel Hill, North Carolina, 27599, United States
Tryon Medical Partners
Charlotte, North Carolina, 28210, United States
Durham Nephrology Associates, PA
Durham, North Carolina, 27704, United States
Duke University School of Medicine - Duke Molecular Physiology Institute
Durham, North Carolina, 27710, United States
Temple University Hospital
Philadelphia, Pennsylvania, 19140, United States
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
South Carolina Nephrology and Hypertension Center, Inc.
Orangeburg, South Carolina, 29118, United States
Vanderbilt University VU
Nashville, Tennessee, 37232, United States
Prolato Clinical Research Center
Houston, Texas, 77054, United States
Privia Medical Group
Houston, Texas, 77095, United States
Hopital Henri Mondor
Créteil, France
Hôpital Bicêtre AP-HP
Le Kremlin-Bicêtre, France
Bichat Hospital
Paris, France
Hopitaux Universitaires Est Parisien - Hopital Tenon
Paris, France
Service de Nephrologie - Hopital Universitaire Necker
Paris, France
Université Paris-Descartes / Hôpital Européen Georges Pompidou
Paris, France
GCM Medical Group, PSC
San Juan, 00917, Puerto Rico
University Hospitals of Leicester NHS Trust - Leicester General Hospital
Leicester, United Kingdom
Barts Health NHS Trust
London, United Kingdom
The Medicines Evaluation Unit
Manchester, United Kingdom
King's College Hospital NHS Foundation Trust - Guthrie Clinic
Southwark, United Kingdom
Related Publications (2)
Egbuna O, Zimmerman B, Manos G, Fortier A, Chirieac MC, Dakin LA, Friedman DJ, Bramham K, Campbell K, Knebelmann B, Barisoni L, Falk RJ, Gipson DS, Lipkowitz MS, Ojo A, Bunnage ME, Pollak MR, Altshuler D, Chertow GM; VX19-147-101 Study Group. Inaxaplin for Proteinuric Kidney Disease in Persons with Two APOL1 Variants. N Engl J Med. 2023 Mar 16;388(11):969-979. doi: 10.1056/NEJMoa2202396.
PMID: 36920755DERIVEDBruggeman LA, Sedor JR, O'Toole JF. Apolipoprotein L1 and mechanisms of kidney disease susceptibility. Curr Opin Nephrol Hypertens. 2021 May 1;30(3):317-323. doi: 10.1097/MNH.0000000000000704.
PMID: 33767059DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Medical Monitor
- Organization
- Vertex Pharmaceuticals Incorporated
Publication Agreements
- PI is Sponsor Employee
- No
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 7, 2020
First Posted
April 9, 2020
Study Start
June 8, 2020
Primary Completion
November 11, 2021
Study Completion
December 9, 2021
Last Updated
September 24, 2025
Results First Posted
July 10, 2023
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will not share
Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent research/clinical-trial-data-sharing.