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
试验速览
- 阶段
- 3 期
- 状态
- 招募中
- 入组人数
- 70
- 试验地点
- 14
- 主要终点
- Change from Baseline in Time to Rise (TTR) Velocity at Week 54
研究概览
简要总结
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
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 7 Years 至 16 Years（Child）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •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
- •Steroid regimen must be anticipated to remain stable.
排除标准
- •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
研究组 & 干预措施
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
干预措施: AOC 1044 (Drug)
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
干预措施: Placebo (Drug)
结局指标
主要结局
Change from Baseline in Time to Rise (TTR) Velocity at Week 54
时间窗: Baseline, Week 54
次要结局
- Change from Baseline in Creatine Kinase (CK) at Week 54(Baseline, Week 54)
- Change from Baseline in 4-Stair Climb (4SC) Velocity at Week 54(Baseline, Week 54)
- Change from Baseline in 10-Meter Walk/Run Test (10MWRT) Velocity at Week 54(Baseline, Week 54)
- Change from Baseline in Stride Velocity 95th Centile (SV95C) at Week 54(Baseline, Week 54)
- Change from Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 54(Baseline, Week 54)
- Change from Baseline in DMD Quality of Life (DMD-QoL) Score at Week 54(Baseline, Week 54)
- Change from Baseline in Patient Global Impression of Severity (PGI-S) at Week 54(Baseline, Week 54)
- Change from Baseline in Caregiver Global Impression of Severity (CaGI-S) at Week 54(Baseline, Week 54)
- Patient Global Impression of Change (PGI-C) at Week 54(Week 54)
- Caregiver Global Impression of Change (CaGI-C) at Week 54(Week 54)
- Change from Baseline in Quantitative Muscle Testing (QMT) at Week 54(Baseline, Week 54)