Health Trials

Phase 2/3 Adaptive Study of VX-147 in Adult and Pediatric Participants With APOL1-Mediated Proteinuric Kidney Disease

ConditionProteinuric Kidney Disease
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age10-65
SponsorVertex Pharmaceuticals Incorporated

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

APOL1 genotype of G1/G1, G2/G2, or G1/G2

Proteinuric kidney disease

Disqualifiers

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

Trial design

Design model

Sequential

Treatments tested in this trial

  • VX-147

    Drug

    Tablets for oral administration.

  • Placebo

    Drug

    Tablets for oral administration.

Treatment groups

466 Participants
are divided into 3 treatment groups
Group A: Part A (Phase 2/3): VX-147Experimental treatment 1 intervention
Group B: Part A (Phase 2/3): PlaceboPlacebo comparator 1 intervention
Group C: Part B (Phase 3): VX-147Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Part A: Percent Change From Baseline in Urine Protein to Creatinine Ratio (UPCR) at Week 48 (Assessed at the Week 48 Interim Analysis)

Time frame
From Baseline to Week 48
2

Part A: Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at Interim Analysis

Time frame
From Baseline Through >= Week 48
3

Part A: eGFR Slope Assessed at Final Analysis

Time frame
From Baseline Through Study Completion (At least 2 years of eGFR data assessed at the final analysis)
4

Part B: Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs)

Time frame
Day 1 Through Study Completion (Approximately 4 Years After the Last Participant Enrolls)

Secondary outcomes

1

Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse events (AEs) and Serious Adverse Events (SAEs)

Time frame
Day 1 Through Study Completion (Approximately 2 Years After the Last Participant Enrolls)
2

Part A: Maximum Plasma Concentration (Cmax) of VX-147

Time frame
Day 1 and Week 40
3

Part A: Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-147

Time frame
Day 1 and Week 40
4

Part A: Observed Pre-dose Plasma Concentration (Ctrough) of VX-147

Time frame
Day 1 up to Week 40

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Vertex Pharmaceuticals Incorporated

Lead sponsor

Source ClinicalTrials.gov
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