An Open-Label Extension Study of EryDex in Patients With Ataxia Telangiectasia Following Participation in Study IEDAT-04-2022 (NEAT)
试验速览
- 阶段
- 3 期
- 状态
- 终止
- 入组人数
- 101
- 试验地点
- 28
- 主要终点
- Incidence of treatment emergent adverse events
研究概览
简要总结
This is an international, multi-center, prospective, open-label, non-comparative study aiming to provide access to treatment with EryDex to ataxia telangiectasia (A-T) patients who completed the IEDAT-04-2022 trial which studied the neurological effects of EryDex on subjects with ataxia telangiectasia (NEAT trial).
详细描述
The IEDAT-05-2024 study aims to provide EryDex (dexamethasone sodium phosphate encapsulated into autologous erythrocytes) treatment to all participants in the IEDAT-04-2022 (NEAT) study, who complete the study assessments, do not -have safety contraindications to continuation of treatment, and who provide informed consent. The open-label extension (OLE) treatment period will be 12 months. Participants will be considered to have completed the study when Visit 14 (Safety Follow-up) has been performed.
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
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •body weight ≥15 kg
- •participation in IEDAT-04-2022 study and its completion, including final efficacy and safety assessments
排除标准
- •safety contraindications for continuation of treatment, as determined by the investigator
- •clinically significant immune impairment that, in the opinion of the Investigator, precludes further treatment with corticosteroids
- •Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years.
- •requiring treatment with a systemic corticosteroid
研究组 & 干预措施
Dexamethasone sodium phosphate
intravenous (IV) infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS)
干预措施: Dexamethasone sodium phosphate (Drug)
结局指标
主要结局
Incidence of treatment emergent adverse events
时间窗: through study completion, up to 14 months
Number of patients presenting at least one treatment emergent adverse event (TEAE)
Incidence of treatment emergent adverse events leading to intervention discontinuation
时间窗: through study completion, up to 14 months
Number of patients presenting at least one treatment emergent adverse event (TEAE) leading to intervention discontinuation
Incidence of serious adverse events
时间窗: through study completion, up to 14 months
Number of patients presenting at least one serious adverse event (SAE)
Number of Participants With Treatment Emergent Adverse Events
时间窗: through study completion, up to approximately 13 months
Number of participants presenting at least one treatment emergent adverse event (TEAE)
Number of Participants With Treatment Emergent Adverse Events Leading to Intervention Discontinuation
时间窗: through study completion, up to approximately 13 months
Number of participants presenting at least one treatment emergent adverse event (TEAE) leading to intervention discontinuation
Number of Participants With Serious Adverse Events
时间窗: through study completion, up to approximately 13 months
Number of participants presenting at least one serious adverse event (SAE)
次要结局
未报告次要终点