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

Sponsor
Avidity Biosciences, Inc.
Study ID
NCT07587242
Phase
PHASE3
Status
Recruiting
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Conditions

  • 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

Eligibility Criteria

Sex
MALE
Age
7 Years - 16 Years
Healthy Volunteers
Not accepted

Interventions

  • AOC 1044 — DRUG
    AOC 1044 will be administered by intravenous (IV) infusion
  • Placebo — DRUG
    Placebo will be administered by intravenous (IV) infusion

Study Details

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

Key Dates

First listed
May 14, 2026
Start date
Aug 31, 2026
Status verified
Aug 2026
Primary completion
May 31, 2029
Completion
Jul 31, 2030

Study Design

Enrollment
70 participants (estimated)
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT

Arms

  • Experimental: AOC 1044
    Participants will receive AOC 1044 intravenously (IV) every 6 weeks (Q6W) during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which the same participants will continue to receive AOC 1044 IV Q6W
  • Placebo Comparator: Placebo
    Participants will receive placebo (saline) IV Q6W during the 54-week double-blind treatment period, followed by a 54-week open-label treatment period in which all participants will then receive AOC 1044 IV Q6W

Primary Outcome Measure

Change from Baseline in Time to Rise (TTR) Velocity at Week 54 [ Time Frame: Baseline, Week 54 ]

Central Contacts

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