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
- 招募中
- 入组人数
- 49
- 试验地点
- 10
- 主要终点
- Safety endpoint : Evaluation of safety and tolerability in this OLE study will be based on the assessment of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), discontinuations due to AEs, changes in vital signs, laboratory parameters, physical and neurological examination findings, and the Columbia Suicide Severity Rating Scale (C-SSRS).
研究概览
简要总结
Safety Objective: To evaluate the safety and tolerability of EryDex in patients with A-T. Exploratory Objective: To evaluate the effect of EryDex on neurological symptoms in patients with A-T.
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Open-Label Extension Study
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 64 years（0-17 Years, 18-64 Years）
- 接受健康志愿者
- 是
入选标准
- •Participant completed the IEDAT-04-2022 study, including final efficacy assessments (Visit 9) and does not present safety contraindications for continuation of treatment as determined by the investigator.
- •Participant’s body weight ≥15 kg.
排除标准
- •Females that are pregnant, or are breastfeeding (for EU countries only), or are of childbearing potential, pregnant, or are breastfeeding (for US and other countries outside of the EU). Females of childbearing potential using adequate birth control as determined by their healthcare provider will be eligible refer to Section 8.
- •Participant has 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.
- •Participant has confirmed hemoglobinopathies, e.g., hemoglobin C disease, sickle cell anemia, hereditary spherocytosis, or thalassemia.
- •Participants with suicidal ideation.
- •Participant requires treatment with a systemic corticosteroid. Treatment with inhaled or intranasal corticosteroids for asthma or allergies, as well as use of topical corticosteroids will be permitted.
- •Participant requires any concomitant medication prohibited by the protocol, including strong inducers or inhibitors of cytochrome P450 3A4 (CYP3A4) (refer to Section 7.8).
结局指标
主要结局
Safety endpoint : Evaluation of safety and tolerability in this OLE study will be based on the assessment of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), discontinuations due to AEs, changes in vital signs, laboratory parameters, physical and neurological examination findings, and the Columbia Suicide Severity Rating Scale (C-SSRS).
Safety endpoint : Evaluation of safety and tolerability in this OLE study will be based on the assessment of treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), discontinuations due to AEs, changes in vital signs, laboratory parameters, physical and neurological examination findings, and the Columbia Suicide Severity Rating Scale (C-SSRS).
次要结局
未报告次要终点
研究者
Dirk Thye
Scientific
Quince Therapeutics S.p.A.