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NCT Number: NCT05312879

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

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.

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Key information

Conditions

Proteinuric Kidney Disease

Age range

10 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

Universite Catholique de Louvain - Pulmonology, Woluwe-Saint-Lambert, Brussels Capital, Belgium

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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/Exclusion criteria will apply.

Treatment and study plan

VX-147

Drug

Tablets for oral administration.

Placebo

Drug

Tablets for oral administration.

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

  5. 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

    Time frame: Day 1 and Week 48

  6. Part B: Percent Change From Baseline in UPCR Over Time

    Time frame: From Baseline Through Study Completion (Approximately 4 Years After the Last Participant Enrolls)

  7. Part B: eGFR Slope Assessment

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

Study contacts

Contact information is provided by the study sponsor or research team.

Medical Information

CONTACT

[email protected]

617-341-6777

Sponsors and collaborators

Lead sponsor

Vertex Pharmaceuticals Incorporated

Industry

Registry information

Official study 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

Acronym: AMPLITUDE

Important dates

Study start
2022
Primary completion
2028
Study completion
2030
First posted
Apr 6, 2022
Registry last updated
Jun 25, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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