A Multi-center, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Neurological Effects of EryDex on Subjects With Ataxia Telangiectasia (NEAT)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 105
- 试验地点
- 36
- 主要终点
- Rescored modified International Cooperative Ataxia Rating Scale (RmICARS)
研究概览
简要总结
This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes), administered by intravenous (IV) infusion once every 28 days, on neurological symptoms of patients with Ataxia Telangiectasia (A-T).
详细描述
The EryDex System (EDS) is a combination product that is used to load dexamethasone sodium phosphate (DSP) into autologous erythrocytes (EryDex) which is infused into the patient.
In the placebo arm, the subjects will receive autologous erythrocytes prepared with the EDS process using a placebo solution.
Upon completion of all screening assessments for eligibility, subjects meeting all selection criteria at baseline will be randomized in a 1:1 fashion to EryDex or placebo. Approximately 86 subjects 6- to 9-years-old, approximately 43 per group, will be randomized. Approximately 20 subjects 10 years of age and above, 10 per treatment group, may also be enrolled.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •Clinical diagnosis of A-T
- •In autonomous gait or is helped by periodic use of a support
- •Genetic confirmation of A-T
- •Body weight ≥15 kg
排除标准
- •Participation in another clinical study
- •Immune impairment
- •History of severe impairment of the immunological system
- •Current neoplastic disease or previous neoplastic disease not in remission for at least 2 years
- •Severe or unstable pulmonary disease
- •Uncontrolled diabetes
- •Current chronic or acute significant renal and/or hepatic impairment
- •Any previous oral or parenteral steroid use within 6 weeks before Baseline. Treatment with inhaled or intranasal steroids for asthma or allergies, as well as use of topical steroids will be permitted
- •A disability that may prevent the subject from completing all study requirements
研究组 & 干预措施
Dexamethasone sodium phosphate
IV infusion of dexamethasone sodium phosphate (DSP) encapsulated in autologous erythrocytes using the EryDex System (EDS)
干预措施: Dexamethasone sodium phosphate (Drug)
Placebo
IV infusion of placebo encapsulated in autologous erythrocytes using the EryDex System (EDS)
干预措施: Placebo (Other)
结局指标
主要结局
Rescored modified International Cooperative Ataxia Rating Scale (RmICARS)
时间窗: Baseline to Visit 9 (approximately 6 months)
Change of the RmICARS from baseline to Visit 9 compared to placebo (6 to 9 years old)
次要结局
- Clinical Global Impression of Change (CGI-C)(Baseline to Visit 9 (approximately 6 months))
- Clinical Global Impression of Severity (CGI-S)(Baseline to Visit 9 (approximately 6 months))