A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 159
- 试验地点
- 68
- 主要终点
- Hand function
研究概览
简要总结
A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous Delpacibart Etedesiran (abbreviated del-desiran, formerly AOC 1001) for the Treatment of Myotonic Dystrophy Type 1
详细描述
The study consists of a Screening Period of up to 6 weeks and 54-week Treatment Period. The anticipated duration is approximately 60 weeks.
Participants will be randomized to receive an intravenous infusion of either del-desiran or placebo at the clinical study site every 8 weeks for a total of 7 doses. The final dose will occur at Week 48, followed by a final assessment at Week 54.
After completion of Week 54 assessments, eligible participants will have the option to enroll into an open label extension (OLE) study, pending regulatory approval.
An Independent Data Monitoring Committee (IDMC) comprised of 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
- 16 Years 至 65 Years（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1
- •Ability to walk independently (orthoses and ankle braces allowed) for at least 10 meters at screening
排除标准
- •Breastfeeding, pregnancy, or intent to become pregnant during the study
- •Unwilling or unable to comply with contraceptive requirements
- •Abnormal lab values, conditions or diseases that would make the participant unsuitable for the study
- •Diabetes that is not adequately controlled
- •History of decompensated heart failure within 3 months of screening. Participants with preexisting pacemaker/ICD are not excluded.
- •Body Mass Index > 35 kg/m2 at Screening
- •Recently treated with an investigational drug or biological agent
- •Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline, whichever is longer, prior to baseline.
- •Note: Additional protocol defined Inclusion and Exclusion criteria apply
研究组 & 干预措施
Del-desiran
Del-desiran (AOC 1001) will be administered seven times
干预措施: AOC 1001 (del-desiran) (Drug)
Placebo
Saline will be administered seven times
干预措施: Placebo (Drug)
结局指标
主要结局
Hand function
时间窗: Through Week 30
video Hand Opening Time (vHOT)
Hand function
时间窗: Through Week 54
video Hand Opening Time (vHOT)
次要结局
- Hand grip strength(Through Week 30)
- Myotonic Dystrophy Type 1 activity and participation scale c(Through Week 30)
- Quantitative Muscle Testing composite score(Through Week 30)
- Hand grip strength(Through Week 54)
- Quantitative Muscle Testing composite score(Through Week 54)
- Myotonic Dystrophy Type 1 activity and participation scale c(Through Week 54)
- 10-Meter Walk/Run Test(Through Week 54)