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

Conditions

APOL1-mediated Proteinuric Kidney Disease (AMKD)

Brief summary

Percent change in UPCR evaluated from baseline at Week 48 (assessed at the IA) • eGFR slope (with ≥48 weeks of eGFR data assessed at the IA and at least 2 years of eGFR data assessed at the final analysis)

Detailed description

Time to composite clinical outcome of a sustained decline of ≥30% from baseline in estimated glomerular filtration rate (eGFR), the onset of ESKD (i.e., maintenance dialysis for ≥28 days, kidney transplantation, or a sustained eGFR of <15 mL/min/1.73 m2), or death. Sustained is defined as confirmation by a second measurement after ≥28 days (assessed at the final analysis)., Safety and tolerability based on adverse events (AEs), clinical laboratory values (i.e., hematology, serum chemistry, urinalysis, coagulation studies), standard 12-lead ECGs, and vital signs, Plasma PK parameters of VX-147, Acceptability of tablet formulation of VX-147 in pediatric subjects using the convenience domain of the Treatment Satisfaction Questionnaire for Medication (TSQM)

Related papers

No related papers found yet.

Interventions

DRUGVX-147

DRUGCoated Placebo Tablet

DRUGVX-147 film-coated tablet

Eligibility

Sex/Gender

All

Age

0 Years to 64 Years

Design outcomes

Primary

Measure	Time frame
Percent change in UPCR evaluated from baseline at Week 48 (assessed at the IA) • eGFR slope (with ≥48 weeks of eGFR data assessed at the IA and at least 2 years of eGFR data assessed at the final analysis)	—

Secondary

Measure	Time frame
Time to composite clinical outcome of a sustained decline of ≥30% from baseline in estimated glomerular filtration rate (eGFR), the onset of ESKD (i.e., maintenance dialysis for ≥28 days, kidney transplantation, or a sustained eGFR of <15 mL/min/1.73 m2), or death. Sustained is defined as confirmation by a second measurement after ≥28 days (assessed at the final analysis)., Safety and tolerability based on adverse events (AEs), clinical laboratory values (i.e., hematology, serum chemistry, urinalysis, coagulation studies), standard 12-lead ECGs, and vital signs, Plasma PK parameters of VX-147, Acceptability of tablet formulation of VX-147 in pediatric subjects using the convenience domain of the Treatment Satisfaction Questionnaire for Medication (TSQM)	—

Measure

Time frame

Time to composite clinical outcome of a sustained decline of ≥30% from baseline in estimated glomerular filtration rate (eGFR), the onset of ESKD (i.e., maintenance dialysis for ≥28 days, kidney transplantation, or a sustained eGFR of <15 mL/min/1.73 m2), or death. Sustained is defined as confirmation by a second measurement after ≥28 days (assessed at the final analysis)., Safety and tolerability based on adverse events (AEs), clinical laboratory values (i.e., hematology, serum chemistry, urinalysis, coagulation studies), standard 12-lead ECGs, and vital signs, Plasma PK parameters of VX-147, Acceptability of tablet formulation of VX-147 in pediatric subjects using the convenience domain of the Treatment Satisfaction Questionnaire for Medication (TSQM)

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Countries

Belgium, France

Outcome results

None listed