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VX-147

Phase 2

Proteinuric Kidney Disease | Small molecule | Nephrology |Vertex Pharmaceuticals Incorporated|Last Updated: Jun 2, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment466
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05312879Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney DiseasePHASE2 RECRUITING 466 — — Mar 30, 2022May 7, 2030Jun 2, 2026318 United States, Belgium +11
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Study Endpoints
Primary Endpoints
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 Endpoints
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
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A (Phase 2/3): VX-147EXPERIMENTALParticipants will be randomized to receive different doses of VX-147. Participants will receive the selected dose of VX-147 for atleast 96 weeks.
Part A (Phase 2/3): PlaceboPLACEBO_COMPARATORParticipants will receive placebo matched to VX-147.
Part B (Phase 3): VX-147EXPERIMENTALParticipants who complete Part A will receive VX-147 for an additional 96 weeks.
Interventions
NameTypeDescription
VX-147DRUGTablets for oral administration.
PlaceboDRUGTablets for oral administration.
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Eligibility Criteria
Age Range10 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites318

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 hy...

Countries:United StatesBelgiumBrazilCanadaColombiaFranceGhanaNetherlandsNigeriaPortugalPuerto RicoSpainUnited Kingdom
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Recent Changes (Last 90 Days)
LOWJun 2, 2026NCT05312879lastUpdatePostDate: changed
LOWJun 2, 2026NCT05312879lastUpdatePostDate: changed
LOWJun 2, 2026NCT05312879lastUpdatePostDate: changed
MEDIUMMay 26, 2026NCT05312879primaryCompletionDate: changed
LOWMay 24, 2026NCT05312879studyFirstPostDate: changed