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
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: AOC 1044, Placebo.
- Who it may be relevant to
- Registry conditions: Muscular Dystrophies, Muscular Dystrophies (Duchenne, Becker, Myotonic Dystrophy), Muscular Disorders, Atrophic, Muscular Disease. Basic parameters: 7 years — 16 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- France, Germany, Italy, Spain
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Global Study With an Open-Label Extension to Evaluate the Efficacy and Safety of Intravenous AOC 1044 (Delpacibart Zotadirsen) for the Treatment of DMD With Gene Mutations Amenable to Exon 44 Skipping
Overview
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
Detailed 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.
Interventions
- Drug AOC 1044
AOC 1044 will be administered by intravenous (IV) infusion - Drug Placebo
Placebo will be administered by intravenous (IV) infusion
Primary outcome measures
- Change from Baseline in Time to Rise (TTR) Velocity at Week 54 [Time frame: Baseline, Week 54]
Secondary outcome measures (11)
- Change from Baseline in Creatine Kinase (CK) at Week 54 [Time frame: Baseline, Week 54]
- Change from Baseline in 4-Stair Climb (4SC) Velocity at Week 54 [Time frame: Baseline, Week 54]
- Change from Baseline in 10-Meter Walk/Run Test (10MWRT) Velocity at Week 54 [Time frame: Baseline, Week 54]
- Change from Baseline in Stride Velocity 95th Centile (SV95C) at Week 54 [Time frame: Baseline, Week 54]
- Change from Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 54 [Time frame: Baseline, Week 54]
- Change from Baseline in DMD Quality of Life (DMD-QoL) Score at Week 54 [Time frame: Baseline, Week 54]
- Change from Baseline in Patient Global Impression of Severity (PGI-S) at Week 54 [Time frame: Baseline, Week 54]
- Change from Baseline in Caregiver Global Impression of Severity (CaGI-S) at Week 54 [Time frame: Baseline, Week 54]
- Patient Global Impression of Change (PGI-C) at Week 54 [Time frame: Week 54]
- Caregiver Global Impression of Change (CaGI-C) at Week 54 [Time frame: Week 54]
- Change from Baseline in Quantitative Muscle Testing (QMT) at Week 54 [Time frame: Baseline, Week 54]
Eligibility criteria
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.
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Quadruple blind
- Primary purpose
- Treatment
Study locations
Germany · 3 centers
- Universitaetsklinikum Essen — Essen
- Universitaetsklinikum Heidelberg — Heidelberg
- Klinikum der Ludwig-Maximilians-Universitaet Muenchen — München
Italy · 2 centers
- Fondazione Serena ETS - Centro Clinico NeMO Milano — Milan
- Fondazione Policlinico Universitario A. Gemelli IRCCS - Universitario Cattolica del Sacro — Roma
France · 1 center
- AP-HP Hospital Armand-Trousseau — Paris
Spain · 1 center
- Hospital Sant Joan de Deu — Barcelona
Identifiers
NCT: NCT07587242 · AOC 1044-CS3