Recruiting
Phase 3

AOC 1044

Sponsor:

Avidity Biosciences, Inc.

Code:

NCT07587242

Conditions

Muscular Dystrophies

Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)

Muscular Disorders, Atrophic

Muscular Disease

Musculoskeletal Diseases

Eligibility Criteria

Sex: Male

Age: 7 - 16

Healthy Volunteers: Not accepted

Interventions

AOC 1044

Placebo

Study Details

Brief summary:

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

Conditions

Muscular Dystrophies

Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy)

Muscular Disorders, Atrophic

Muscular Disease

Musculoskeletal Diseases

Study ID

NCT07587242

Start date

Sep 1, 2026

Status verified date

Nov, 2026

Completion date

Jul, 2030

Anticipated

Primary completion date

May, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: Male

Age: 7 - 16

Healthy Volunteers: Not accepted

Key Inclusion Criteria:

  • 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
  • On a stable regimen of corticosteroids (including Vamolorone) for at least 6 months prior to Day 1. Steroid regimen must be anticipated to remain stable.

Key Exclusion Criteria:

  • 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
  • If on any of the following treatments (growth hormone, testosterone or givinostat), participants must be on a stable regimen and must plan to maintain it for the duration of the study. Participants will be excluded if regimen stability prior to informed consent is as follows:
  • Less than 1 month, for growth hormone and/or testosterone
  • Less than 6 months for givinostat

Study Design

Enrollment

70 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

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

Interventions

AOC 1044

AOC 1044 will be administered by intravenous (IV) infusion

Placebo

Placebo will be administered by intravenous (IV) infusion

Primary outcome measure

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

Central Contacts and Locations

Central contacts

Avidity Bioscience, Inc., A Novartis Company

1-888-669-6682Novartis.email@novartis.com

Locations

London Health Sciences Centre - Victoria Hospital

Recruiting

London, Ontario, Canada, N6A 5W9

More Information

Sponsor

Avidity Biosciences, Inc.

Last update posted

Nov 10, 2026

Last verified

Nov, 2026

Keywords

  • AOC
  • AOC 1044
  • AOC 1044-CS3
  • AOC 1044-CS1
  • AOC 1044-CS2
  • EXPLORE44
  • EXPLORE44-OLE
  • SAFARI
  • SAFARI44
  • SAFARI 44
  • Avidity
  • Avidity Biosciences
  • Exon Skipping Therapy
  • Avidity Biosciences Inc., A Novartis Company
  • del-zota
  • DMD

Trial information was received from ClinicalTrials.gov and was last updated on 2026-11-11. This information was provided to ClinicalTrials.gov by Avidity Biosciences, Inc. on 2026-11-10. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.