Experimental drug targets genetic cause of alport syndrome in tiny pilot
NCT ID NCT05448755
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.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Great Ormond Street Hospital
London, WC1N 3JH, United Kingdom
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Monash Medical Center
Clayton, Victoria, 3168, Australia
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Royal Children's Hospital
Parkville, Victoria, 3051, Australia
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Royal Free Hospital
London, NW3 2QG, United Kingdom