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New drug aims to slow kidney damage in rare alport syndrome

NCT ID NCT07523581

Disease control Sponsor: Eloxx Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 16, 2026 · Updated 5 times

Summary

This study tests a drug called exaluren in 24 people aged 12 and older with Alport syndrome caused by specific genetic errors. The drug is given as a daily shot for 32 weeks. Half the group starts exaluren right away, while the other half gets a placebo first and then switches to exaluren. The goal is to see if exaluren can improve kidney health by looking at tiny changes in kidney tissue and protein levels in urine.

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

About 24 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Jun 2026

An estimate. Start dates often move.

Expected to finish

Dec 2027

An estimate. End dates often move.

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

12 years and older

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.

Inclusion Criteria: * A confirmed diagnosis of X-linked or autosomal recessive Alport Syndrome with a documented nonsense mutation of COL4A5 in a male or nonsense mutation of COL4A3 or COL4A4 (male or female) * eGFR\>45 ml/min/1.73 m2 * Urinary protein based on two spot urine collections \[urine protein/creatinine ratio (UPCR) ≥ 500 mg/g\] * Stable regimen of ACEi/ARB for at least 12 weeks before Day 1 Exclusion Criteria: * History of any organ transplantation * Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 1.5 times the upper limit of normal (ULN) will be excluded * History of dialysis * Acute kidney injury within 4 weeks before screening * Active dizziness

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Conditions

The condition(s) this trial relates to.

Alport syndrome autosomal recessive Alport syndrome hereditary nephritis X-linked Alport syndrome

As listed by the trial registrant

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

Alport syndrome, autosomal recessive Alport syndrome, X-linked

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    12 sites in 2 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Alder Hey Children's NHS Foundation Trust

    NOT_YET_RECRUITING

    Liverpool, L12 2AP, United Kingdom

  • Cleveland Clinic

    NOT_YET_RECRUITING

    Cleveland, Ohio, 44195, United States

  • Denver Nephrologists PC, Colorado Kidney Care PC

    RECRUITING

    Denver, Colorado, 80220, United States

  • Great Ormond Street Hospital

    RECRUITING

    London, WC1N 3JH, United Kingdom

  • Hackensack University Medical Center

    NOT_YET_RECRUITING

    Hackensack, New Jersey, 07601, United States

  • Renal Associates, P.A.

    NOT_YET_RECRUITING

    San Antonio, Texas, 78109, United States

  • Royal Free Hospital

    RECRUITING

    London, NW3 2QG, United Kingdom

  • Royal Manchester Children's Hospital

    NOT_YET_RECRUITING

    Manchester, M13 9WL, United Kingdom

  • Stanford University

    NOT_YET_RECRUITING

    Stanford, California, 94305, United States

  • University of California, Los Angeles

    NOT_YET_RECRUITING

    Los Angeles, California, 90095, United States

    Contact Email: •••••@•••••

  • University of Minnesota

    NOT_YET_RECRUITING

    Minneapolis, Minnesota, 55455, United States

  • University of Pennsylvania

    NOT_YET_RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

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Other studies related to the condition(s) this trial covers.