Experimental kidney drug for alport syndrome falls short in early trial

NCT ID NCT02855268

Terminated Disease control Sponsor: Genzyme, a Sanofi Company Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This phase 2 trial tested a weekly injection of lademirsen in 43 adults with Alport syndrome, a genetic condition that damages kidneys. The goal was to see if the drug could slow the decline in kidney function. The study was terminated early, so the results are limited and it's unclear if lademirsen is safe or effective.

What this could mean

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

Active substance

lademirsen (a drug injection)

What this could lead to

If it worked, lademirsen could slow kidney function loss in people with Alport syndrome, delaying the need for dialysis or transplant.

What could go wrong

The trial was terminated early with only 43 participants, so we don't have enough data to know if it's safe or effective. Early-phase results often don't lead to approved treatments.

Disclaimer Read more

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.

Alport syndrome hereditary nephritis kidney disorder nephritis

As listed by the trial registrant

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

ALPORT SYNDROME

Contacts and locations

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Locations

Columbia University Medical Center_Investigational Site Number :8400004

New York, New York, 10032, United States
Investigational Site Number :0360001

Parkville, Victoria, 3050, Australia
Investigational Site Number :0360002

Nedlands, Western Australia, 6009, Australia
Investigational Site Number :0360003

Herston, Queensland, 4029, Australia
Investigational Site Number :1560001

Beijing, 100034, China
Investigational Site Number :1560002

Beijing, 100730, China
Investigational Site Number :1560004

Guangzhou, 510080, China
Investigational Site Number :2500001

Paris, 75015, France
Investigational Site Number :2500002

Toulouse, 31403, France
Investigational Site Number :7240001

Barcelona, Barcelona [Barcelona], 08025, Spain
Investigational Site Number :7240002

Madrid / Madrid, Madrid, Comunidad de, 28040, Spain
Investigational Site Number :7240003

Granada, 18014, Spain
Investigational Site Number :7240004

Barcelona, Barcelona [Barcelona], 08035, Spain
Investigational Site Number :7240005

Córdoba, Andalusia, 14004, Spain
Investigational Site Number :8260001

London, London, City of, NW3 2QG, United Kingdom
Investigational Site Number :8260002

Nottingham, Nottinghamshire, NG5 1PB, United Kingdom
Investigational Site Number :8260003

Newcastle upon Tyne, NE7 7DN, United Kingdom
Investigational Site Number :8400002

Los Angeles, California, 90024, United States
The Cleveland Clinic Foundation_Investigational Site Number :8400001

Cleveland, Ohio, 44195, United States
Uniklinik Köln, Innere Medizin II - Nephrologie, Rheumatologie, Diabetologie und Allgemeine Innere Medizin_Investigational Site Number :2760001

Cologne, 50937, Germany
University of Minnesota Childrens' Hospital_Investigational Site Number :8400003

Minneapolis, Minnesota, 55454, United States
University of Utah_Investigational Site Number :8400005

Salt Lake City, Utah, 84132, United States
Universitätsmedizin Göttingen, Klinik für Nephrologie und Rheumatologie_Investigational Site Number :2760002

Göttingen, 37075, Germany