A Phase 2 Open-label Study to Evaluate the Pharmacodynamics and Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants With Mutations Amenable to Exon 44 Skipping
试验速览
- 阶段
- 2 期
- 状态
- 进行中（未招募）
- 入组人数
- 39
- 试验地点
- 18
- 主要终点
- Incidence of Treatment Emergent Adverse Events (TEAEs)
研究概览
简要总结
AOC 1044-CS2 (EXPLORE44-OLE) is an Open-label Study to Evaluate the Long-Term Safety and Tolerability of AOC 1044 Administered Intravenously to DMD Participants with Mutations Amenable to Exon 44 Skipping.
详细描述
AOC 1044-CS2 (EXPLORE44-OLE) is an open label, extension study to Part B of AOC 1044-CS1 (EXPLORE44). AOC 1044-CS2 is designed to evaluate the long-term safety, tolerability, pharmacokinetics, and exploratory efficacy of AOC 1044.
All participants who enroll in AOC 1044-CS2 will receive active treatment. The treatment period is 2 years with IV dosing every 6 weeks.
Once participants have completed active treatment, they will be followed through a 3-month safety follow-up period. The sponsor may extend active treatment beyond 2 years at a future timepoint.
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
盲法说明
Randomization from the parent study, AOC 1044-CS1 will remain blinded.
入排标准
- 年龄范围
- 7 Years 至 27 Years（Child, Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Rollover Participants:
- •Satisfactorily completed AOC 1044-CS1 (EXPLORE44) as determined by the Investigator and Sponsor
- •No significant tolerability issues with AOC 1044
- •De novo Participants:
- •Aged 7 to 27 years, inclusive, at the time of informed consent
- •Clinical diagnosis of DMD or clear onset of DMD symptoms at or before the age of 6 years
- •Confirmation of DMD gene mutation amenable to exon 44 skipping
- •Weight ≥ 23 kg
- •Ambulatory or non-ambulatory
- •Ambulatory participants: LVEF ≥50% and FVC≥50%
- •Non-ambulatory participants: LVEF ≥45% and FVC≥40%
- •PUL 2.0 entry item A ≥3
- •If on corticosteroids, stable dose for 30 days before screening and throughout the study
排除标准
- •Rollover Participants:
- •Presence of any new condition or worsening of existing condition that could affect a participant's safety or ability to comply with study procedures
- •De novo Participants:
- •Serum hemoglobin < lower limit of normal
- •Uncontrolled hypertension or diabetes
- •Prior treatment with any cell or gene therapy
- •Prior treatment with another exon 44 skipping agent within 6 months prior to informed consent
- •Recently treated with an investigational drug
- •History of multiple drug allergies
研究组 & 干预措施
AOC 1044 Multiple Dose Levels
AOC 1044 will be IV infused every 6 weeks for approximately 2 years.
干预措施: AOC 1044 (Drug)
结局指标
主要结局
Incidence of Treatment Emergent Adverse Events (TEAEs)
时间窗: Through study completion (approximately 2 years)
次要结局
- Change from baseline in serum creatine kinase concentration at Study Weeks 24, 48, and 102(Through study completion (approximately 2 years))