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New drug targets genetic kidney disease in small trial

NCT ID NCT06794996

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Aug 21, 2026 · Updated 2 times

Summary

This study tests a drug called inaxaplin in 42 people with a genetic form of kidney disease linked to the APOL1 gene. The goal is to see if the drug can reduce protein in the urine, a sign of kidney damage, and to check for side effects. Participants take the drug as tablets, and the study is open-label, meaning everyone knows they are getting the drug.

What this could mean

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

Active substance
inaxaplin (also called IXP or VX-147)
What this could lead to
If successful, this could point toward a new treatment option for people with a genetic form of kidney disease, potentially slowing kidney damage.
What could go wrong
This is a small, early-phase trial with only 42 participants and no placebo group, so results may not be definitive. The drug may not prove effective or could have side effects.

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

42 people

The number who actually took part.

Started

Feb 2025

Finished

Jul 2026

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 67 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: * Participant has an APOL1 genotype of G1/G1, G2/G2, or G1/G2 obtained with a Vertex designated investigational clinical study assay * Estimated Glomerular Filtration Rate (eGFR) of greater than or equal to (≥) 25 milliliter per minute (mL/min)/1.73m\^2 at screening Key Exclusion Criteria: * Evidence of Focal Segmental Glomerulosclerosis (FSGS) with a known cause other than due to APOL1 risk variants * Uncontrolled hypertension Other protocol defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

focal segmental glomerulosclerosis 4, susceptibility to

As listed by the trial registrant

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

Proteinuric kidney disease

Contacts and locations

Locations

  • CTR Oakwater, LLC

    Orlando, Florida, 32806, United States

  • Carolina Nephrology, PA

    Spartanburg, South Carolina, 29306, United States

  • Columbia Nephrology Associates, PA

    Columbia, South Carolina, 29203, United States

  • DaVita Kidney Care - Las Vegas

    Las Vegas, Nevada, 89107, United States

  • Dallas Nephrology Associates - Dallas Landry Office

    Dallas, Texas, 75246, United States

  • Dallas Renal Group - 1

    Dallas, Texas, 75237, United States

  • Dallas Renal Group - 2

    Dallas, Texas, 75230, United States

  • Duke University Hospital - Children's Health Center

    Durham, North Carolina, 27710, United States

  • Eastern Nephrology Associates - New Bern Office

    New Bern, North Carolina, 28562, United States

  • Foundation for Sickle Cell Disease Research, LLC

    Hollywood, Florida, 33023, United States

  • Georgia Nephrology

    Lawrenceville, Georgia, 30046, United States

  • Georgia Nephrology - Decatur

    Decatur, Georgia, 30030, United States

  • Javara Inc. /Privia Medical Group Georgia, LLC - Savannah

    Savannah, Georgia, 31406, United States

  • Javara Inc./Privia Medical Group Georgia, LLC - Fayetteville

    Fayetteville, Georgia, 30214, United States

  • Javara Inc./Privia Medical Group, LLC MidAtlantic-Silver Spring, MD

    Silver Spring, Maryland, 20901, United States

  • LCMC Health

    Metairie, Louisiana, 70006, United States

  • Lewis Katz School of Medicine at Temple University - Section of Nephrology

    Philadelphia, Pennsylvania, 19140, United States

  • NYC Health + Hospital/Harlem

    New York, New York, 10037, United States

  • Nephrology Associates, P.C.

    Columbus, Mississippi, 39705, United States

  • Nephrology Consultants, LLC

    Huntsville, Alabama, 35805, United States

  • Nephrology and Hypertension Associates, LTD

    Tupelo, Mississippi, 38801, United States

  • North Carolina Nephrology P.A. - Cary Office

    Cary, North Carolina, 27511, United States

  • Northwest Louisiana Nephrology, LLC - Shreveport

    Shreveport, Louisiana, 71101, United States

  • Ochsner Medical Center - New Orleans - Nephrology

    New Orleans, Louisiana, 70121, United States

  • Prolato Clinical Research Center

    Houston, Texas, 77054, United States

  • Renal Associates of Baton Rouge

    Baton Rouge, Louisiana, 70808, United States

  • Renal Associates, LLC

    Columbus, Georgia, 31904, United States

  • Rutgers Doctors Office Center

    Newark, New Jersey, 07103, United States

  • Saint Michael's Medical Center - Peter Ho Memorial Clinic

    Newark, New Jersey, 07102, United States

  • Schiff Center for Liver Diseases

    Miami, Florida, 33136, United States

  • Scott Research, Inc.

    Laurelton, New York, 11413, United States

  • South Florida Research Institute

    Lauderdale Lakes, Florida, 33313, United States

  • Tks Research, P.L.L.C.

    Norfolk, Virginia, 23504, United States

  • UNC Clinical and Translational Research Center

    Chapel Hill, North Carolina, 27599-7155, United States

  • UTSW Medical Center - Nephrology

    Dallas, Texas, 75390, United States

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