Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease

NCT05312879 · tracked via the Priya Life Science UK tracker

Sponsor

Vertex Pharmaceuticals Incorporated

Phase

Phase 2/3

Started

2022-03-30

Last updated

2026-09-09

Condition(s) studied

Proteinuric Kidney Disease

Investigational drug(s) / intervention(s)

VX-147Placebo

VX-147: Tablets for oral administration.

Placebo: Tablets for oral administration.

Study summary

The purpose of this study is to evaluate the efficacy, safety, tolerability, and pharmacokinetics (PK) of VX-147 in adult and pediatric participants with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.

Eligibility

Sex

ALL

Min age

10 Years

Max age

65 Years

Healthy volunteers

Key Inclusion Criteria: Part A: * APOL1 genotype of G1/G1, G2/G2, or G1/G2 * Proteinuric kidney disease Part B: \- Completion of Treatment Period in Part A and no permanent discontinuation of study drug. Key Exclusion Criteria: Part A: * Solid organ or bone marrow transplant * Uncontrolled hypertension * History of diabetes mellitus * Known underlying cause of kidney disease including but not limited to sickle cell disease Part B: * ESKD (End Stage Kidney Disease) as defined in the protocol. * Any lab abnormality that may pose a safety risk to the participant, as judged by the investigator. Other protocol defined Inclusion/Exclusion criteria will apply.

Primary outcome measure(s)

Part A: Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis) — From Baseline to Week 48
Part A: Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at Interim Analysis — From Baseline Through >= Week 48
Part A: eGFR Slope Assessed at Final Analysis — From Baseline Through Study Completion (At least 2 years of eGFR data assessed at the final analysis)
Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs) — Day 1 Through Study Completion (Approximately 4 Years After the Last Participant Enrolls)

Trial sites (318)

Facility	City	Region	Status
Alabama Kidney Research	Alabaster	Alabama	Recruiting
EmVenio Research - Mobile Unit - Birmingham	Birmingham	Alabama	Recruiting
Cardiology, P.C.	Birmingham	Alabama	Completed
Children's Hospital of Alabama	Birmingham	Alabama	Recruiting
The Kirklin Clinic - Nephrology	Birmingham	Alabama	Recruiting
Nephrology Associates	Fairhope	Alabama	Recruiting
Heart Center Research LLC	Huntsville	Alabama	Recruiting
Nephrology Consultants, LLC	Huntsville	Alabama	Recruiting
Renal Associates of Alabama, LLC	Montgomery	Alabama	Recruiting
Arizona Kidney Disease and Hypertension Center - Banner Desert Office	Mesa	Arizona	Withdrawn
Arizona Kidney Disease and Hypertension Center - Thomas Office	Phoenix	Arizona	Recruiting
Phoenix Children's Hospital, Inc. - Nephrology	Phoenix	Arizona	Completed
CHI St. Vincent Heart Clinic Arkansas- Cardiology and Medicine Clinic - Little Rock	Little Rock	Arkansas	Completed
UAMS Medical Center - Pulmonology	Little Rock	Arkansas	Completed
The Medical Research Group - Saint Agnes Medical Center	Fresno	California	Recruiting
Amicis Research Center - Balboa	Granada Hills	California	Recruiting
Kidney Care Center of North Valley	Granada Hills	California	Recruiting
Eastside Clinical Research Associates	Los Angeles	California	Recruiting
Kaiser Permanente Los Angeles Medical Center	Los Angeles	California	Recruiting
Cedars-Sinai Medical Center	Los Angeles	California	Recruiting
UCLA Division of Nephrology	Los Angeles	California	Completed
UCI Center for Clinical Research	Orange	California	Completed
Kaiser Permanente Sacramento Medical Center	Sacramento	California	Recruiting
Helen Diller Medical Center at Parnassus Heights	San Francisco	California	Recruiting
UCLA Lundquist Institute at Harbor-UCLA Medical Center	Torrance	California	Completed
Kidney and Hypertension Center	Victorville	California	Completed
Greater Hartford Nephrology	Bloomfield	Connecticut	Recruiting
Nephrology & Hypertension Associates, PC	Middlebury	Connecticut	Completed
Yale University - Yale Nephrology Clinical Research Clinic	New Haven	Connecticut	Recruiting
Children's National Hospital - Nephrology	Washington D.C.	District of Columbia	Recruiting
The George Washington University - GW Medical Faculty Associates	Washington D.C.	District of Columbia	Recruiting
Veterans Affairs Medical Center - Washington D.C	Washington D.C.	District of Columbia	Recruiting
Bay Pines Foundation	Bay Pines	Florida	Recruiting
Nova Clinical Research, LLC - Bradenton - 59th St	Bradenton	Florida	Recruiting
Clinical Research of Brandon, LLC	Brandon	Florida	Completed
AMR-Miami	Coral Gables	Florida	Completed
Horizon Research Group - Coral Gables	Coral Gables	Florida	Completed
South Florida Nephrology Research	Coral Springs	Florida	Recruiting
Delray Physician Care Center	Delray Beach	Florida	Recruiting
North Florida/South Georgia Veterans Health System	Gainesville	Florida	Recruiting

+ 278 more sites — see the full list on the official registry below.

More Vertex Pharmaceuticals Incorporated trials in the UK

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A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy Active, not recruiting

A Phase 1/2 Study of VX-522 in Participants With Cystic Fibrosis (CF) Active, not recruiting

Evaluation of Efficacy and Safety of VX-993 for Pain Associated With Diabetic Peripheral Neuropathy Recruiting

Evaluation of Long-term Safety and Efficacy of ELX/TEZ/IVA in Cystic Fibrosis Participants 12 Months of Age and Older Active, not recruiting

Other trials for the same condition

A Phase 2 Study of MZE829 in Adults With APOL1 Kidney Disease Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05312879 on ClinicalTrials.gov ↗ ← All trials in the UK