A Phase 2 Extension Study to Evaluate the Long-Term Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 37
- 试验地点
- 16
- 主要终点
- Number and severity of treatment-emergent adverse events (TEAEs)
研究概览
简要总结
AOC 1001-CS2 (MARINA-OLE) (NCT05479981) is a Phase 2 extension of the AOC 1001-CS1 (MARINA) (NCT05027269) study to evaluate the safety, tolerability, efficacy, pharmacokinetics and pharmacodynamics of multiple-doses of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) patients
详细描述
This study will continue to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and efficacy of AOC 1001 in participants that enrolled in the randomized, placebo-controlled, First-In-Human Phase 1/2 AOC 1001-CS1 (MARINA) (NCT05027269) clinical study.
Participants from AOC 1001-CS1 (NCT05027269) are eligible to enroll in AOC 1001-CS2 (NCT05479981) if they have satisfactorily completed AOC 1001-CS1 (NCT05027269).
After completion of Q8W (once every 8 weeks) Visit 6 assessments, participants will have the option to enroll in another AOC 1001 open-label extension (OLE) study - AOC 1001-CS4 (HARBOR-OLE) (NCT07008469). Participants who do not enroll in an AOC 1001 OLE study will be monitored for safety for a total of 8 weeks from the last dose of study drug. Total duration on study is up to 37 months (3 years 1 month).
As of September 2024, the dosing regimen was updated to every 8 weeks.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
盲法说明
Participants will receive one blinded treatment at Day 43. Treatment assignment on this day will be based on treatment received in AOC 1001-CS1 (NCT05027269) so that participants who did not receive active drug on Day 43 in AOC 1001-CS1 (NCT05027269) will receive active drug on Day 43 in AOC 1001-CS2 (NCT05479981).
入排标准
- 年龄范围
- 18 Years 至 66 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Completion of AOC 1001-CS1 (MARINA) study with satisfactory compliance and no significant tolerability issues
排除标准
- •Pregnancy, intent to become pregnant, or active breastfeeding
- •Unwilling or unable to continue to comply with contraceptive requirements
- •Any new conditions or worsening of existing conditions that in the opinion of the investigator or sponsor would make the participant unsuitable for the study or could interfere with participation or completion of the study
研究组 & 干预措施
AOC 1001 (with Placebo at Day 43)
AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.
干预措施: AOC 1001 (Drug)
AOC 1001 (with Placebo at Day 43)
AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant received AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive blinded placebo treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.
干预措施: Placebo (Drug)
AOC 1001
AOC 1001 will initially be administered quarterly. On Day 43 patients will receive an additional dose. Treatment assignment will be based on treatment received in AOC 1001-CS1. If participant did not receive AOC 1001 on Day 43 in AOC 1001-CS1, participant will receive AOC 1001 treatment on Day 43 in AOC 1001-CS2. Beginning in September 2024, AOC 1001 will be administered every 8 weeks.
干预措施: AOC 1001 (Drug)
结局指标
主要结局
Number and severity of treatment-emergent adverse events (TEAEs)
时间窗: Through study completion
次要结局
- Change and percentage change from baseline in DMPK mRNA knockdown(Through Day 183)
- Change and percentage change from baseline in Spliceopathy(Through Day 183)
- Change and percentage change from baseline in DMPK mRNA knockdown(Through Day 183)
- Change and percentage change from baseline in Spliceopathy(Through Day 183)
- Plasma pharmacokinetic (PK) parameters(Up to Day 729)
- AOC 1001 levels in muscle tissue(Through Day 183)