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.
Brief Title
Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1- Mediated Proteinuric Kidney Disease
Central Contacts
Central Contact Role
Contact
Central Contact Phone
617-341-6777
Central Contact Email
medicalinfo@vrtx.com
Completion Date
Completion Date Type
Estimated
Conditions
Proteinuric Kidney Disease
Eligibility Criteria
Key Inclusion Criteria:
* APOL1 genotype of G1/G1, G2/G2, or G1/G2
* Proteinuric kidney disease
Key Exclusion Criteria:
* 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
Other protocol defined Inclusion/Exclusion criteria apply.
* APOL1 genotype of G1/G1, G2/G2, or G1/G2
* Proteinuric kidney disease
Key Exclusion Criteria:
* 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
Other protocol defined Inclusion/Exclusion criteria apply.
Inclusion Criteria
Inclusion Criteria:
* APOL1 genotype of G1/G1, G2/G2, or G1/G2
* Proteinuric kidney disease
Inclusion/
* APOL1 genotype of G1/G1, G2/G2, or G1/G2
* Proteinuric kidney disease
Inclusion/
Gender
All
Gender Based
false
Keywords
APOL1-mediated kidney disease (AMKD)
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
65 Years
Minimum Age
10 Years
NCT Id
NCT05312879
Org Class
Industry
Org Full Name
Vertex Pharmaceuticals Incorporated
Org Study Id
VX21-147-301
Overall Status
Recruiting
Phases
Phase 2
Phase 3
Primary Completion Date
Primary Completion Date Type
Estimated
Official Title
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
Primary Outcomes
Outcome Measure
Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis)
Outcome Time Frame
From Baseline at Week 48
Outcome Measure
Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at the Week 48 Interim Analysis
Outcome Time Frame
From Baseline Through >= Week 48
Outcome Measure
eGFR Slope Assessed at Study Completion
Outcome Time Frame
From Baseline Through Study Completion (Approximately 2 Years After the Last Participant Enrolls)
Secondary Ids
Secondary Id
2024-515633-15-00
Secondary Outcomes
Outcome Time Frame
From Baseline Through Study Completion (Approximately 2 Years After the Last Participant Enrolls)
Outcome Measure
Time to Composite Clinical Outcome of a Sustained Decline of >=30 Percent (%) in eGFR, the Onset of end-stage Kidney Disease or Death
Outcome Time Frame
Day 1 Through Study Completion (Approximately 2 Years After the Last Participant Enrolls)
Outcome Measure
Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs)
Outcome Time Frame
Day 1 and Week 40
Outcome Measure
Maximum Plasma Concentration (Cmax) of VX-147
Outcome Time Frame
Day 1 and Week 40
Outcome Measure
Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-147
Outcome Time Frame
Day 1 up to Week 40
Outcome Measure
Observed Pre-dose Plasma Concentration (Ctrough) of VX-147
Outcome Time Frame
Day 1 and Week 48
Outcome Measure
Acceptability Tablet Formulation of VX-147 in Pediatric Participants using the Convenience Domain of the Treatment Satisfaction Questionnaire for Medication (TSQM) Version 1.4
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
65
Minimum Age Number (converted to Years and rounded down)
10
Investigators
Investigator Type
Principal Investigator
Investigator Name
Michael Ross
Investigator Email
michael.ross@einsteinmed.edu
Investigator Phone
718-430-8768