RecruitingPhase 2/3

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

United States, Belgium, Brazil466 participantsStarted 2022-03-30

Plain-language 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.

Who can participate

Age range

10 Years – 65 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Part A: Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis)

Timeframe: From Baseline to Week 48

Part A: Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at Interim Analysis

Timeframe: From Baseline Through >= Week 48

Part A: eGFR Slope Assessed at Final Analysis

Timeframe: 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)

Timeframe: Day 1 Through Study Completion (Approximately 4 Years After the Last Participant Enrolls)

Trial details

NCT IDNCT05312879

SponsorVertex Pharmaceuticals Incorporated

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-06-02

Contact for this trial

Medical Information

617-341-6777 medicalinfo@vrtx.com

View on ClinicalTrials.gov More Proteinuric Kidney Disease trials