Phase 2a Study of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis

June 17, 2023 updated by: Vertex Pharmaceuticals Incorporated

A Phase 2a, Open-label, Single-arm, 2-Part Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of VX-147 in Adults With APOL1-mediated Focal Segmental Glomerulosclerosis

This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-147 in participants with apolipoprotein L1 (APOL1)-mediated focal segmental glomerulosclerosis (FSGS).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

16

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Créteil, France
        • Hopital Henri Mondor
      • Le Kremlin-Bicêtre, France
        • Hôpital Bicêtre AP-HP
      • Paris, France
        • Bichat Hospital
      • Paris, France
        • Hopitaux Universitaires Est Parisien - Hopital Tenon
      • Paris, France
        • Service de Nephrologie - Hopital Universitaire Necker
      • Paris, France
        • Université Paris-Descartes / Hôpital Européen Georges Pompidou
  • Puerto Rico
      • San Juan, Puerto Rico, 00917
        • GCM Medical Group, PSC
  • United Kingdom
      • Leicester, United Kingdom
        • University Hospitals of Leicester NHS Trust - Leicester General Hospital
      • London, United Kingdom
        • Barts Health NHS Trust
      • Manchester, United Kingdom
        • The Medicines Evaluation Unit
      • Southwark, United Kingdom
        • King's College Hospital NHS Foundation Trust - Guthrie Clinic
  • United States
    • Alabama
      • Birmingham, Alabama, United States, 35294
        • University of Alabama at Birmingham - The Kirklin Clinic
    • California
      • San Diego, California, United States, 92123
        • California Institute of Renal Research
    • District of Columbia
      • Washington, District of Columbia, United States, 20060
        • Howard University Hospital
      • Washington, District of Columbia, United States, 20037
        • The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension
    • Florida
      • Miami, Florida, United States, 33150
        • Kidney and Hypertension Specialists of Miami
      • Winter Park, Florida, United States, 32789
        • Florida Premier Research Institute
    • Georgia
      • Atlanta, Georgia, United States, 30329
        • Children's Healthcare of Atlanta
      • Atlanta, Georgia, United States, 30310
        • Morehouse School of Medicine, Grady Memorial Hospital
      • Lawrenceville, Georgia, United States, 30046
        • Georgia Nehphrology
      • Macon, Georgia, United States, 31201
        • Central Georgia Kidney Specialists PC
    • Louisiana
      • Baton Rouge, Louisiana, United States, 70808
        • Renal Associates of Baton Rouge
      • New Orleans, Louisiana, United States, 70121
        • Ochsner Medical Center - New Orleans
    • Massachusetts
      • Boston, Massachusetts, United States, 02215
        • Beth Israel Deaconess Medical Center
      • Boston, Massachusetts, United States, 02118
        • Boston Medical Center
      • Springfield, Massachusetts, United States, 01107
        • Renal and Transplant Associates of New England, PC
    • Michigan
      • Kalamazoo, Michigan, United States, 49007
        • Paragon Health, PC d/b/a Nephrology Center, PC
    • Mississippi
      • Tupelo, Mississippi, United States, 38801
        • Nephrology and Hypertension Associates, LTD
    • Missouri
      • Saint Louis, Missouri, United States, 63136
        • St Louis Kidney Care
    • Nevada
      • Las Vegas, Nevada, United States, 89106
        • Nevada Kidney Disease and Hypertension Centers
    • New York
      • Brooklyn, New York, United States, 11203
        • SUNY Downstate
      • Buffalo, New York, United States, 14201
        • Urban Family Practice
      • New York, New York, United States, 10029
        • Icahn School of Medicine at Mount Sinai
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • UNC Kidney Center Division of Nephrology & Hypertension
      • Charlotte, North Carolina, United States, 28210
        • Tryon Medical Partners
      • Durham, North Carolina, United States, 27704
        • Durham Nephrology Associates, Pa
      • Durham, North Carolina, United States, 27710
        • Duke University School of Medicine - Duke Molecular Physiology Institute
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19140
        • Temple University Hospital
      • Pittsburgh, Pennsylvania, United States, 15224
        • Children's Hospital of Pittsburgh of UPMC
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina
      • Orangeburg, South Carolina, United States, 29118
        • South Carolina Nephrology and Hypertension Center, Inc.
    • Tennessee
      • Nashville, Tennessee, United States, 37232
        • Vanderbilt University VU
    • Texas
      • Houston, Texas, United States, 77054
        • Prolato Clinical Research Center
      • Houston, Texas, United States, 77095
        • Privia Medical Group

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 63 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • APOL1 genotype of G1/G1, G2/G2, or G1/G2
  • FSGS diagnosed by kidney biopsy

Key Exclusion Criteria:

  • Evidence of non-APOL1-mediated FSGS
  • Participants with known sickle cell disease
  • Solid organ or Bone marrow transplant

Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: VX-147
All participants received VX-147 at a dosage of 15 mg once daily (qd) for 2 weeks and VX-147 at a dosage of 45 mg qd for 11 weeks. Part A was enrolled in 2 cohorts: Cohort 1 and Cohort 2. Cohort 1 included participants with urine protein to creatinine ratio (UPCR) approximately greater than or equal to (≥) 3 g/g (± 10%) and less than (<) 10 g/g and estimated glomerular filtration rate (eGFR) approximately ≥30 mL/min/1.73 m2 (± 10%). Cohort 2 included participants with UPCR approximately ≥0.8 g/g (± 10%) and <2.7 g/g and eGFR approximately ≥30 mL/min/1.73 m2.
Drug: VX-147
Tablets for oral administration.
Other Names:
  • IXP

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Percent Change From Baseline in UPCR
Time Frame: From Baseline up to Week 13
From Baseline up to Week 13

Secondary Outcome Measures

Outcome Measure
Time Frame
Safety and Tolerability as Assessed by Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time Frame: From Baseline up to Week 17
From Baseline up to Week 17
Maximum Observed Concentration (Cmax) of VX-147
Time Frame: Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5
Pre-dose and at 0.25, 0.5, 1, 2, 4, and 12 hours post-dose on Day 1 and Week 5
Observed Pre-dose Concentration (Ctrough) of VX-147
Time Frame: Pre-dose on Day 8, 15, Week 3, 5, 9 and 13
Pre-dose on Day 8, 15, Week 3, 5, 9 and 13
Area Under the Concentration Time-curve From 0 to 24 Hours (AUC0-24hr) of VX-147
Time Frame: Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5
Pre-dose and at 0.25, 0.5, 1, 2, 4, 12 and 24 hours Post-dose on Week 5

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Vertex Pharmaceuticals Incorporated

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 8, 2020

Primary Completion (Actual)

November 11, 2021

Study Completion (Actual)

December 9, 2021

Study Registration Dates

First Submitted

April 7, 2020

First Submitted That Met QC Criteria

April 7, 2020

First Posted (Actual)

April 9, 2020

Study Record Updates

Last Update Posted (Actual)

July 10, 2023

Last Update Submitted That Met QC Criteria

June 17, 2023

Last Verified

June 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VX19-147-101
  • 2020-000185-42 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD 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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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