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

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ClinicalTrials.gov Identifier: NCT05312879
Recruitment Status : Recruiting
First Posted : April 6, 2022
Last Update Posted : May 9, 2024
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Study Description
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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.

Condition or disease Intervention/treatment Phase
Proteinuric Kidney Disease Drug: VX-147 Drug: Placebo Phase 2 Phase 3

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 466 participants
Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : March 30, 2022
Estimated Primary Completion Date : May 2026
Estimated Study Completion Date : June 2026

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Kidney Diseases

Arms and Interventions
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Arm Intervention/treatment
Experimental: Phase 2: VX-147
Participants will be randomized to receive different dose levels of VX-147.
 Drug: VX-147
Tablets for oral administration.
Placebo Comparator: Phase 2: Placebo
Participants will receive placebo matched to VX-147.
 Drug: Placebo
Tablets for oral administration.
Experimental: Phase 3: VX-147
Participants will receive VX-147 with the dose to be based on the outcome of Phase 2.
 Drug: VX-147
Tablets for oral administration.
Placebo Comparator: Phase 3: Placebo
Participants will receive placebo matched to VX-147.
 Drug: Placebo
Tablets for oral administration.


Outcome Measures
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Primary Outcome Measures :
  1. 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 at Week 48 ]
  2. Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at the Week 48 Interim Analysis [ Time Frame: From Baseline Through >= Week 48 ]
  3. eGFR Slope Assessed at Study Completion [ Time Frame: From Baseline Through Study Completion (Approximately 2 Years After the Last Participant Enrolls) ]

Secondary Outcome Measures :
  1. Time to Composite Clinical Outcome of a Sustained Decline of >=30 Percent (%) in eGFR, the Onset of end-stage Kidney Disease or Death [ Time Frame: From Baseline Through Study Completion (Approximately 2 Years After the Last Participant Enrolls) ]
  2. 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) ]
  3. Maximum Plasma Concentration (Cmax) of VX-147 [ Time Frame: Day 1 and Week 40 ]
  4. Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-147 [ Time Frame: Day 1 and Week 40 ]
  5. Observed Pre-dose Plasma Concentration (Ctrough) of VX-147 [ Time Frame: Day 1 up to Week 40 ]
  6. 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 ]

Eligibility Criteria
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Ages Eligible for Study:   10 Years to 65 Years   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05312879


Contacts
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Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com

Locations
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Sponsors and Collaborators
Vertex Pharmaceuticals Incorporated
More Information
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Responsible Party: Vertex Pharmaceuticals Incorporated
ClinicalTrials.gov Identifier: NCT05312879    
Other Study ID Numbers: VX21-147-301
2021-004762-35 ( EudraCT Number )
First Posted: April 6, 2022    Key Record Dates
Last Update Posted: May 9, 2024
Last Verified: January 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No
Plan Description: Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/independent-research/clinical-trial-data-sharing

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Kidney Diseases
Urologic Diseases
Female Urogenital Diseases
 Female Urogenital Diseases and Pregnancy Complications
Urogenital Diseases
Male Urogenital Diseases