RecruitingNCT07587242ClinicalTrials.gov record

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

Muscular Dystrophies · Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy) · Muscular Disorders, Atrophic · Muscular Disease · Musculoskeletal Diseases · Neuromuscular Diseases (NMD) · Nervous System Diseases · Genetic Diseases · X-Linked · Hereditary · Neonatal Disease · Duchene Muscular Dystrophy · Congenital · DMD

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THE QUICK READ

What this study involves

The registry says this study is recruiting. Individual locations may have different availability.

Study type: Interventional
Phase: Phase 3
Age range: 7 years - 16 years
Sex eligible: Male
Healthy volunteers: Not accepted
Participants: 70 Estimated

These are starting points, not an eligibility decision. The research team must review the full requirements.

About the research

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

Read the full description

The study consists of a Screening Period of up to 44 Days, a Randomized 54 Week Treatment Period, a 54 Week Open Label Extension (OLE), and a Follow-Up Period of 6 weeks. Participants will be randomized to receive an intravenous infusion of either delpacibart-zotadirsen or placebo at the clinical study site every 6 weeks for a total of 9 doses. After completion of the randomized treatment period, all participants may enter the OLE portion of the study consisting of 9 doses of AOC 1044 regardless of group assignment in the randomized period. The final dose will occur at Week 102, followed by a final assessment at Week 108 and a safety follow-up visit at Week 114. An Independent Data Monitoring Committee (IDMC) comprising members independent and external to the Sponsor will review safety, tolerability, and efficacy (as needed) data of this study at regular intervals.

Who may be able to participate?

The list below is the sponsor’s full registry wording. It can include conditions, prior treatment, test results and other requirements. A match in search does not mean you qualify.

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

Understand screening

Treatment, groups and participation

The registry may describe treatments and study duration without a complete visit calendar. Ask the study team for the exact schedule, procedures, risks, costs and alternatives. Keep your usual care plan unless your treating clinician tells you otherwise.

Assignment: Random assignment
Masking: Quadruple masked

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

Drug: AOC 1044

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

Drug: Placebo

AOC 1044 DRUG

AOC 1044 will be administered by intravenous (IV) infusion

Placebo DRUG

Placebo will be administered by intravenous (IV) infusion

Prepare your questions

What researchers are measuring

Outcome measures describe what is being studied. They are not proof that a treatment works.