A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1
试验速览
- 阶段
- 3 期
- 状态
- Enrolling By Invitation
- 入组人数
- 199
- 试验地点
- 44
- 主要终点
- TEAEs
研究概览
简要总结
A Global Phase 3 Open-Label Extension Study to Assess the Long-Term Safety, Tolerability, and Efficacy 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 either 4-weeks or 8-weeks depending on the prior parent trial, and up to a 4-year Treatment Period. The anticipated duration is 50 months and 2 weeks (4 years and 2.5 months).
Participants will receive an intravenous infusion of del-desiran at the clinical study site every 8 weeks for a total of 7 doses per year. The final dose will occur at Year 4, Visit 7, followed by a final assessment 8 weeks after the last dose.
An additional subgroup of de novo participants will also be included in a Fixed-Dose PK cohort.
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
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 16 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •FROM A PARENT STUDY
- •Key Inclusion Criteria:
- •Completion of any prior AOC 1001 studies with satisfactory completion of dosing and follow-up assessments and satisfactory compliance with the protocol requirements of the parent study, as determined by the Investigator.
排除标准
- •Breastfeeding, pregnancy, or intent to become pregnant during the study.
- •Unwilling to comply with contraceptive requirements.
- •Any new conditions or worsening of existing condition that in the opinion of the Investigator would make the participant unsuitable for the study.
- •FIXED-DOSE PK COHORT
- •Key Inclusion Criteria:
- •Clinical and genetic diagnosis of DM1 and clinically significant hand myotonia.
- •Key Exclusion Criteria:
- •Breastfeeding, pregnancy, or intent to become pregnant during the study
- •Unwilling 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
- •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 per year for up to 4 years.
干预措施: Del-desiran (AOC 1001) (Drug)
结局指标
主要结局
TEAEs
时间窗: From enrollment to the end of treatment at 4 years
Incidence of treatment emergent adverse events
次要结局
未报告次要终点