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

To evaluate VX-147 in patients with APOL1-mediated focal segmental glomerulosclerosis To evaluate the safety and efficacy of VX-147 and to assess the impact of VX-147, on top of standard of care, on kidney function and proteinuria in people with AMKD To evaluate two doses of VX-147 for 12 weeks to select a dose for Phase III and subsequently evaluate the efficacy and safety of the single, selected dose in the Phase III portion of the study 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. To evaluate different doses of IXP to select a dose for Ph3 and subsequently evaluate the efficacy and safety of the selected IXP dose in the Ph3 portion of the trial.

CT.gov Identifier
NCT05312879
EudraCT Identifier
2021-004762-35
CTIS:
2024-515633-15-00
Sponsor
Vertex Pharmaceuticals
Collaborator
N/A
Study Contact Information
+1 617 341 6100
Recruiting
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Study Details

Diseases
APOL1 Mediated Kidney Disease (AMKD)
Study Drug
Inaxaplin
See More
Undisclosed - FDA approved drug
Genes
APOL1
Study Dates
Mar 2022 - Jun 2028
Sex
Female & Male
Age
10 - 65 Years
Inclusion and Exclusion Criteria
Inclusion Criteria:
  • Part A:
  • APOL1 genotype of G1/G1, G2/G2, or G1/G2Proteinuric kidney diseasePart B:
  • - Completion of Treatment Period in Part A and no permanent discontinuation of study drug.
Exclusion Criteria:
  • Part A:
  • Solid organ or bone marrow transplantUncontrolled hypertensionHistory of diabetes mellitusKnown underlying cause of kidney disease including but not limited to sickle cell diseasePart 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.

Protocol Summary

To evaluate VX-147 in patients with APOL1-mediated focal segmental glomerulosclerosis

To evaluate the safety and efficacy of VX-147 and to assess the impact of VX-147, on top of standard of care, on kidney function and proteinuria in people with AMKD

To evaluate two doses of VX-147 for 12 weeks to select a dose for Phase III and subsequently evaluate the efficacy and safety of the single, selected dose in the Phase III portion of the study

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.

To evaluate different doses of IXP to select a dose for Ph3 and subsequently evaluate the efficacy and safety of the selected IXP dose in the Ph3 portion of the trial.

Study Locations

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