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

September 22, 2025 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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
16

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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 D.C., District of Columbia, United States, 20060
        • Howard University Hospital
      • Washington D.C., 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
      • St 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years to 61 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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 8, 2020

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
November 11, 2021

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 9, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 7, 2020

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 7, 2020

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 9, 2020

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 24, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
September 22, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
September 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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