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New drug targets genetic kidney disease in small trial

NCT ID NCT04340362

Disease control Sponsor: Vertex Pharmaceuticals Incorporated Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tested a new drug called VX-147 in 16 adults with a specific genetic form of kidney disease (APOL1-mediated FSGS). The goal was to see if the drug could reduce protein in the urine, a sign of kidney damage, and to check its safety. The drug is taken by mouth and aims to control the disease, not cure it.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
VX-147 (a drug taken by mouth)
What this could lead to
If it works, this could point toward a first targeted treatment for a specific genetic form of kidney disease.
What could go wrong
This is a very small, early-phase trial with only 16 people. It may not show clear benefit, and side effects are still being studied.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

16 people

The number who actually took part.

Started

Jun 2020

Finished

Dec 2021

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 to 65 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

focal segmental glomerulosclerosis focal segmental glomerulosclerosis 4, susceptibility to

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Glomerulosclerosis, focal segmental

Contacts and locations

Locations

  • Barts Health NHS Trust

    London, United Kingdom

  • Beth Israel Deaconess Medical Center

    Boston, Massachusetts, 02215, United States

  • Bichat Hospital

    Paris, France

  • Boston Medical Center

    Boston, Massachusetts, 02118, United States

  • California Institute of Renal Research

    San Diego, California, 92123, United States

  • Central Georgia Kidney Specialists PC

    Macon, Georgia, 31201, United States

  • Children's Healthcare of Atlanta

    Atlanta, Georgia, 30329, United States

  • Children's Hospital of Pittsburgh of UPMC

    Pittsburgh, Pennsylvania, 15224, United States

  • Duke University School of Medicine - Duke Molecular Physiology Institute

    Durham, North Carolina, 27710, United States

  • Durham Nephrology Associates, PA

    Durham, North Carolina, 27704, United States

  • Florida Premier Research Institute

    Winter Park, Florida, 32789, United States

  • GCM Medical Group, PSC

    San Juan, 00917, Puerto Rico

  • Georgia Nehphrology

    Lawrenceville, Georgia, 30046, United States

  • Hopital Henri Mondor

    Créteil, France

  • Hopitaux Universitaires Est Parisien - Hopital Tenon

    Paris, France

  • Howard University Hospital

    Washington D.C., District of Columbia, 20060, United States

  • Hôpital Bicêtre AP-HP

    Le Kremlin-Bicêtre, France

  • Icahn School of Medicine at Mount Sinai

    New York, New York, 10029, United States

  • Kidney and Hypertension Specialists of Miami

    Miami, Florida, 33150, United States

  • King's College Hospital NHS Foundation Trust - Guthrie Clinic

    Southwark, United Kingdom

  • Medical University of South Carolina

    Charleston, South Carolina, 29425, United States

  • Morehouse School of Medicine, Grady Memorial Hospital

    Atlanta, Georgia, 30310, United States

  • Nephrology and Hypertension Associates, LTD

    Tupelo, Mississippi, 38801, United States

  • Nevada Kidney Disease and Hypertension Centers

    Las Vegas, Nevada, 89106, United States

  • Ochsner Medical Center - New Orleans

    New Orleans, Louisiana, 70121, United States

  • Paragon Health, PC d/b/a Nephrology Center, PC

    Kalamazoo, Michigan, 49007, United States

  • Privia Medical Group

    Houston, Texas, 77095, United States

  • Prolato Clinical Research Center

    Houston, Texas, 77054, United States

  • Renal Associates of Baton Rouge

    Baton Rouge, Louisiana, 70808, United States

  • Renal and Transplant Associates of New England, PC

    Springfield, Massachusetts, 01107, United States

  • SUNY Downstate

    Brooklyn, New York, 11203, United States

  • Service de Nephrologie - Hopital Universitaire Necker

    Paris, France

  • South Carolina Nephrology and Hypertension Center, Inc.

    Orangeburg, South Carolina, 29118, United States

  • St Louis Kidney Care

    St Louis, Missouri, 63136, United States

  • Temple University Hospital

    Philadelphia, Pennsylvania, 19140, United States

  • The George Washington University Medical Faculty Associates - Kidney Disease & Hypertension

    Washington D.C., District of Columbia, 20037, United States

  • The Medicines Evaluation Unit

    Manchester, United Kingdom

  • Tryon Medical Partners

    Charlotte, North Carolina, 28210, United States

  • UNC Kidney Center Division of Nephrology & Hypertension

    Chapel Hill, North Carolina, 27599, United States

  • University Hospitals of Leicester NHS Trust - Leicester General Hospital

    Leicester, United Kingdom

  • University of Alabama at Birmingham - The Kirklin Clinic

    Birmingham, Alabama, 35294, United States

  • Université Paris-Descartes / Hôpital Européen Georges Pompidou

    Paris, France

  • Urban Family Practice

    Buffalo, New York, 14201, United States

  • Vanderbilt University VU

    Nashville, Tennessee, 37232, United States

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