Recruiting NCT05312879

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

Phase II / Phase III Interventional Proteinuric Kidney Disease

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied: The protocol lists: VX-147, Placebo.
Who it may be relevant to: Registry conditions: Proteinuric Kidney Disease. Basic parameters: 10 years — 65 years · All.
What needs checking: Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place: United States, Belgium, Brazil, Canada, Colombia +8
Next step: Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease

Overview

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.

Interventions

Drug VX-147
Tablets for oral administration.
Drug Placebo
Tablets for oral administration.

Primary outcome measures

Part A: Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis) [Time frame: From Baseline to Week 48]
Part A: Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at Interim Analysis [Time frame: From Baseline Through >= Week 48]
Part A: eGFR Slope Assessed at Final Analysis [Time frame: 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) [Time frame: Day 1 Through Study Completion (Approximately 4 Years After the Last Participant Enrolls)]

Secondary outcome measures (7)

Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs) [Time frame: Day 1 Through Study Completion (Approximately 2 Years After the Last Participant Enrolls)]
Part A: Maximum Plasma Concentration (Cmax) of VX-147 [Time frame: Day 1 and Week 40]
Part A: Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-147 [Time frame: Day 1 and Week 40]
Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-147 [Time frame: Day 1 up to Week 40]
Part A: Acceptability Tablet Formulation of VX-147 in Pediatric Participants using the Convenience Domain of the Treatment Satisfaction Questionnaire for Medication (TSQM) Version 1.4 [Time frame: Day 1 and Week 48]
Part B: Percent Change From Baseline in UPCR Over Time [Time frame: From Baseline Through Study Completion (Approximately 4 Years After the Last Participant Enrolls)]
Part B: eGFR Slope Assessment [Time frame: Day 1 Through Study Completion (Approximately 4 Years After the Last Participant Enrolls)]

Eligibility criteria

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.

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation: Randomized
Model: Sequential
Masking: Quadruple blind
Primary purpose: Treatment