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Evaluating VX-147 for kidney disease linked to APOL1 genetics

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

Phase2; Phase3 Interventional Vertex Pharmaceuticals Incorporated · NCT05312879

This study is testing a new medication called VX-147 to see if it can help adults and kids with kidney disease caused by APOL1 genetics feel better and improve their kidney function.

Quick facts

PhasePhase2; Phase3
Study typeInterventional
Enrollment466 (estimated)
Ages10 Years to 65 Years
SexAll
SponsorVertex Pharmaceuticals Incorporated Industry-sponsored
Locations318 sites (Alabaster, Alabama and 317 other locations)
Trial IDNCT05312879 on ClinicalTrials.gov

What this trial studies

This study aims to assess the efficacy, safety, tolerability, and pharmacokinetics of VX-147 in both adult and pediatric patients suffering from APOL1-mediated proteinuric kidney disease. Participants will be randomly assigned to receive either VX-147 or a placebo, allowing researchers to compare outcomes between the two groups. The study is designed to adapt based on interim results, which may enhance its effectiveness and efficiency in evaluating the treatment's impact on kidney function.

Who should consider this trial

Good fit: Ideal candidates include adults and children with specific APOL1 genotypes (G1/G1, G2/G2, or G1/G2) who are diagnosed with proteinuric kidney disease.

Not a fit: Patients with solid organ or bone marrow transplants, uncontrolled hypertension, diabetes mellitus, or known underlying causes of kidney disease may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve kidney health and quality of life for patients with APOL1-mediated proteinuric kidney disease.

How similar studies have performed: While this approach is focused on a specific genetic condition, similar studies targeting genetic factors in kidney disease have shown promise, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Alabaster, Alabama and 317 other locations

+268 more sites — see ClinicalTrials.gov for the full list.

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Proteinuric Kidney DiseaseAPOL1-mediated kidney disease
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.