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

Phase 2

Recruiting

• Conditions: Proteinuric Kidney Disease
• Interventions: Drug: VX-147; Drug: Placebo

• Registration Number: NCT05312879
• Lead Sponsor: Vertex Pharmaceuticals Incorporated

Brief 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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-03-28
• Study First Submitted QC: 2022-03-28
• Study Start: 2022-03-30
• Study First Posted: 2022-04-06
• Status Verified: 2024-09
• Last Update Submitted: 2025-07-09
• Last Update Posted: 2025-07-15
• Primary Completion: 2026-05-31
• Study Completion: 2026-06-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 466

Inclusion Criteria

• APOL1 genotype of G1/G1, G2/G2, or G1/G2
• Proteinuric kidney disease

Key

Exclusion Criteria

• 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

Other protocol defined Inclusion/Exclusion criteria apply.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Phase 3: VX-147	VX-147	Participants will receive VX-147 with the dose to be based on the outcome of Phase 2.
Phase 3: Placebo	Placebo	Participants will receive placebo matched to VX-147.
Phase 2: VX-147	VX-147	Participants will be randomized to receive different dose levels of VX-147.
Phase 2: Placebo	Placebo	Participants will receive placebo matched to VX-147.

Primary Outcome Measures

Name	Time	Method
eGFR Slope Assessed at Study Completion	From Baseline Through Study Completion (Approximately 2 Years After the Last Participant Enrolls)
Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis)	From Baseline at Week 48
Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at the Week 48 Interim Analysis	From Baseline Through >= Week 48

Secondary Outcome Measures

Name	Time	Method
Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs)	Day 1 Through Study Completion (Approximately 2 Years After the Last Participant Enrolls)
Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-147	Day 1 and Week 40
Observed Pre-dose Plasma Concentration (Ctrough) of VX-147	Day 1 up to Week 40
Acceptability Tablet Formulation of VX-147 in Pediatric Participants using the Convenience Domain of the Treatment Satisfaction Questionnaire for Medication (TSQM) Version 1.4	Day 1 and Week 48
Time to Composite Clinical Outcome of a Sustained Decline of >=30 Percent (%) in eGFR, the Onset of end-stage Kidney Disease or Death	From Baseline Through Study Completion (Approximately 2 Years After the Last Participant Enrolls)
Maximum Plasma Concentration (Cmax) of VX-147	Day 1 and Week 40

Trial Locations

Locations (312)

Alabama Kidney Research; 🇺🇸 Alabaster, Alabama, United States

EmVenio Research - Mobile Unit - Birmingham; 🇺🇸 Birmingham, Alabama, United States

Cardiology, P.C.; 🇺🇸 Birmingham, Alabama, United States

Children's Hospital of Alabama; 🇺🇸 Birmingham, Alabama, United States

The Kirklin Clinic - Nephrology; 🇺🇸 Birmingham, Alabama, United States

Nephrology Associates; 🇺🇸 Fairhope, Alabama, United States

Heart Center Research LLC; 🇺🇸 Huntsville, Alabama, United States

Nephrology Consultants, LLC; 🇺🇸 Huntsville, Alabama, United States

Renal Associates of Alabama, LLC; 🇺🇸 Montgomery, Alabama, United States

Phoenix Children's Hospital, Inc. - Nephrology; 🇺🇸 Phoenix, Arizona, United States

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