Proteinuric Kidney Disease
Conditions
Keywords
APOL1-mediated kidney disease (AMKD)
Brief summary
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
DRUGVX-147
Tablets for oral administration.
DRUGPlacebo
Tablets for oral administration.
Sponsors
Vertex Pharmaceuticals Incorporated
Study design
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
10 Years to 65 Years
Healthy volunteers
No
Inclusion 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/
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Part A: Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis) | From Baseline to Week 48 |
| Part A: Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at Interim Analysis | From Baseline Through >= Week 48 |
| Part A: eGFR Slope Assessed at Final Analysis | 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) | Day 1 Through Study Completion (Approximately 4 Years After the Last Participant Enrolls) |
Secondary
| Measure | Time frame |
|---|---|
| Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs) | Day 1 Through Study Completion (Approximately 2 Years After the Last Participant Enrolls) |
| Part A: Maximum Plasma Concentration (Cmax) of VX-147 | Day 1 and Week 40 |
| Part A: Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-147 | Day 1 and Week 40 |
| Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-147 | 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 | Day 1 and Week 48 |
| Part B: Percent Change From Baseline in UPCR Over Time | From Baseline Through Study Completion (Approximately 4 Years After the Last Participant Enrolls) |
| Part B: eGFR Slope Assessment | Day 1 Through Study Completion (Approximately 4 Years After the Last Participant Enrolls) |
Countries
Belgium, Brazil, Canada, Colombia, France, Ghana, Netherlands, Nigeria, Portugal, Puerto Rico, Spain, United Kingdom, United States
Contacts
CONTACTMedical Information
Outcome results
None listed