Health Trials

A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping

ConditionsMuscular DystrophiesMuscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)Muscular Disorders, AtrophicMuscular DiseaseMusculoskeletal DiseasesNeuromuscular Diseases (NMD)Nervous System DiseasesGenetic DiseasesX-LinkedHereditaryNeonatal DiseaseDuchene Muscular DystrophyCongenitalDMD
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexMale
Age7-16
SponsorAvidity Biosciences, Inc.

About this trial

A Randomized, Double-blind, Placebo-controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Intravenous AOC 1044 for the treatment of Duchenne Muscular Dystrophy (DMD) with Gene Mutations Amenable to Exon 44 Skipping

Eligibility criteria

Qualifiers

Ambulatory males with clinical and genetic diagnosis of DMD

Acceptable genetic test confirming dystrophin gene mutation amenable to exon 44 skipping

7 to 16 years of age at time of consent

TTR and NSAA assessment completed within the protocol specified parameters at Screening

Disqualifiers

Previous treatment cell or gene therapy.

Treatment with another oligonucleotide within 6 months of informed consent (not including COVID-19 RNA vaccines).

Lab values outside of the protocol specified range at Screening

Less than 1 month, for growth hormone and/or testosterone

Trial design

Treatments tested in this trial

  • AOC 1044
  • Placebo

Treatment groups

70 Participants
are divided into 2 treatment groups

Locations

This trial has no locations

Sponsors and collaborators

Avidity Biosciences, Inc.

Lead sponsor

Source ClinicalTrials.gov