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Experimental drug targets genetic cause of alport syndrome in tiny pilot

NCT ID NCT05448755

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 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jun 27, 2026

Summary

This phase 2 pilot study tested a drug called ELX-02 in 3 adults with Alport syndrome caused by specific 'nonsense' mutations. The goal was to see if the drug is safe and if it can reduce protein in the urine, a sign of kidney damage. Participants received daily injections for 8 weeks and were followed for 12 more weeks.

What this could mean

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

Active substance
ELX-02
What this could lead to
If successful, ELX-02 could offer a treatment option for people with Alport syndrome caused by specific genetic errors, potentially slowing kidney damage.
What could go wrong
This was a very small pilot study (only 3 participants) and results are not yet known. The drug may not reduce proteinuria or may cause side effects. Larger trials are needed.

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

3 people

The number who actually took part.

Started

Nov 2022

Finished

Sep 2023

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

6 to 30 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.

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) * The nonsense mutation should be UAG or UGA * eGFR\>60 ml/min/1.73 m2 (based on CKD-EPI for ages ≥18 and Schwartz formula for participants \<18) * Urinary protein based on two spot urine collections \[urine protein/creatinine ratio (UPCR) ≥ 500 mg/g\] * Stable regimen of ACEi/ARB for at least 4 weeks before screening (unless there is a contraindication) Exclusion Criteria: * History of any organ transplantation * Mutation consistent with autosomal dominant Alport Syndrome * Liver disease characterized by cirrhosis or portal hypertension. Participants with alanine aminotransferase (ALT), aspartate aminotransferase (AST), and/or a total bilirubin 3.0 times the upper limit of normal (ULN) will be excluded * History of congestive heart failure diagnosed clinically or with documented left ventricular ejection fraction (LVEF) ≤ 40% * History of dialysis

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Conditions

The condition(s) this trial relates to.

Alport syndrome hereditary nephritis

As listed by the trial registrant

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

Alport syndrome

Contacts and locations

Locations

  • Great Ormond Street Hospital

    London, WC1N 3JH, United Kingdom

  • Monash Medical Center

    Clayton, Victoria, 3168, Australia

  • Royal Children's Hospital

    Parkville, Victoria, 3051, Australia

  • Royal Free Hospital

    London, NW3 2QG, United Kingdom

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