Experimental drug targets genetic cause of alport syndrome in tiny pilot

NCT ID NCT05448755

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

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.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for ALPORT SYNDROME are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

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

More trials for these conditions

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