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

Phase 3RecruitingNCT05312879

Vertex Pharmaceuticals Incorporated466 enrolled

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

466

Conditions

Proteinuric Kidney Disease

Interventions

VX-147DRUG

Tablets for oral administration.

PlaceboDRUG

Tablets for oral administration.

Eligibility

Sex: ALLMin age: 10 YearsMax age: 65 Years

Medical Language ↔ Plain English

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.

Locations (318)

Outcomes

Primary Outcomes

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 Outcomes

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

Central Contacts

Medical Information

CONTACT

617-341-6777 medicalinfo@vrtx.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

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