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Phase 2/3 Adaptive Study of VX-147 in Adults With APOL1- Mediated Proteinuric Kidney Disease

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05312879
Recruitment Status : Recruiting
First Posted : April 6, 2022
Last Update Posted : November 25, 2022
Sponsor:
Information provided by (Responsible Party):
Vertex Pharmaceuticals Incorporated

Tracking Information
First Submitted Date  ICMJE March 28, 2022
First Posted Date  ICMJE April 6, 2022
Last Update Posted Date November 25, 2022
Actual Study Start Date  ICMJE March 30, 2022
Estimated Primary Completion Date May 2026   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 28, 2022)
  • 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 ]
  • Estimated Glomerular Filtration Rate (eGFR) Slope Assessed at the Week 48 Interim Analysis [ Time Frame: From Baseline Through >= Week 48 ]
  • eGFR Slope Assessed at Study Completion [ Time Frame: From Baseline Through Study Completion (Approximately 2 Years After the Last Participant Enrolls) ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: March 28, 2022)
  • 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) ]
  • 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) ]
  • Maximum Observed Plasma Concentration (Cmax) of VX-147 [ Time Frame: Day 1 and Week 12 ]
  • Area Under the Concentration Versus Time Curve During a Dosing Interval (AUCtau) of VX-147 [ Time Frame: Day 1 and Week 12 ]
  • Observed Pre-dose Plasma Concentration (Ctrough) of VX-147 [ Time Frame: Day 1 up to Week 48 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Phase 2/3 Adaptive Study of VX-147 in Adults With APOL1- Mediated Proteinuric Kidney Disease
Official Title  ICMJE A Phase 2/3 Adaptive, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VX-147 in Subjects Aged 18 Years and Older With APOL1-mediated Proteinuric Kidney Disease
Brief Summary The purpose of this study is to evaluate the efficacy, safety, tolerability and pharmacokinetics (PK) of VX-147 in participants aged 18 years and older with apolipoprotein L1 (APOL1)-mediated proteinuric kidney disease.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Sequential Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Proteinuric Kidney Disease
Intervention  ICMJE
  • Drug: VX-147
    Tablets for oral administration.
  • Drug: Placebo
    Tablets for oral administration.
Study Arms  ICMJE
  • Experimental: Phase 2: VX-147
    Participants will be randomized to receive different dose levels of VX-147.
    Intervention: Drug: VX-147
  • Placebo Comparator: Phase 2: Placebo
    Participants will receive placebo matched to VX-147.
    Intervention: Drug: Placebo
  • Experimental: Phase 3: VX-147
    Participants will receive VX-147 with the dose to be based on the outcome of Phase 2.
    Intervention: Drug: VX-147
  • Placebo Comparator: Phase 3: Placebo
    Participants will receive placebo matched to VX-147.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: March 28, 2022)
466
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2026
Estimated Primary Completion Date May 2026   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
  • Diagnosis of diabetes mellitus
  • Known underlying cause of kidney disease including but not limited to sickle cell disease

Other protocol defined Inclusion/Exclusion criteria apply.

Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 60 Years   (Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Information 617-341-6777 medicalinfo@vrtx.com
Listed Location Countries  ICMJE Canada,   France,   Puerto Rico,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT05312879
Other Study ID Numbers  ICMJE VX21-147-301
2021-004762-35 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
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
Current Responsible Party Vertex Pharmaceuticals Incorporated
Original Responsible Party Same as current
Current Study Sponsor  ICMJE Vertex Pharmaceuticals Incorporated
Original Study Sponsor  ICMJE Same as current
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Vertex Pharmaceuticals Incorporated
Verification Date March 2022

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP