Hope for duchenne: new drug targets rare gene mutation in phase 3 trial

NCT ID NCT07587242

Recruiting now Disease control Sponsor: Avidity Biosciences, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 12, 2026 · Updated 6 times

Summary

This study tests an experimental drug called AOC 1044 for boys with Duchenne muscular dystrophy (DMD) who have a specific genetic change (exon 44 skipping). About 70 boys aged 7 to 16 who can still walk will receive either the drug or a placebo. The main goal is to see if the drug helps them stand up faster after 54 weeks of treatment.

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Conditions

The condition(s) this trial relates to.

atrophic muscular disease congenital Duchenne muscular dystrophy hereditary disease Infant, Newborn, Diseases inherited muscle tissue disorder Muscular Disorders, Atrophic muscular dystrophy musculoskeletal system disorder myopathy myotonic dystrophy nervous system disorder neuromuscular disease perinatal disease X-linked disease

As listed by the trial registrant

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

CONGENITAL DMD DUCHENE MUSCULAR DYSTROPHY GENETIC DISEASES HEREDITARY MUSCULAR DISEASE MUSCULAR DISORDERS, ATROPHIC MUSCULAR DYSTROPHIES MUSCULAR DYSTROPHIES (DUCHENNE, BECKER, MYOTONIC DYSTROPHY) MUSCULOSKELETAL DISEASES NEONATAL DISEASE NERVOUS SYSTEM DISEASES NEUROMUSCULAR DISEASES (NMD) X-LINKED

Contacts and locations

Locations

AP-HP Hospital Armand-Trousseau
RECRUITING

Paris, France
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro Cuore
RECRUITING

Roma, Italy
Fondazione Serena ETS - Centro Clinico NeMO Milano
RECRUITING

Milan, Italy
Hospital Sant Joan de Deu
RECRUITING

Barcelona, Spain
Hospital Universitario La Paz
RECRUITING

Madrid, Spain
Hospital Viamed Santa Angela De la Cruz - FutureMeds Spain
RECRUITING

Seville, Spain
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
RECRUITING

München, Germany
Royal Victoria Infirmary
RECRUITING

Newcastle, United Kingdom
Universitaetsklinikum Essen
RECRUITING

Essen, Germany
Universitaetsklinikum Heidelberg
RECRUITING

Heidelberg, Germany

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