Evaluations of Effects of Intra-Erythrocyte Dexamethasone Sodium Phosphate on Neurological Symptoms in Ataxia Teleangiectasia Patients
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 22
- 试验地点
- 2
- 主要终点
- Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population
研究概览
简要总结
The study has the aim to evaluate the improvement in CNS symptoms measured by International Co-operative Ataxia Rating Scale (ICARS) in patients with ataxia teleangectasia (AT), during a period of treatment with ERY-DEX (dexamethasone sodium phosphate ex vivo encapsulated into human autologous erythrocytes).
详细描述
This is a multi-centre, single arm, open label, 6 months, phase II study to evaluate the effect of ERY-DEX in improving Central Nervous System (CNS) symptoms in patients with Ataxia Teleangectasia (AT). The study consists of a screening period (max duration of 30 days) and a treatment period (duration 6 months).
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
- 3 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •neurological signs of AT
- •patients in autonomous gait or helped by a support
- •proven molecular diagnosis of AT
- •Males and females aged > 3 years
- •Body weight >15 kg
- •Plasma levels of Lymphocytes CD4+/mm3 > 500 (for patients aged 3-6 years) or > 200 (older than 6 years)
- •written IC to participate.
排除标准
- •Current or previous neoplastic disease
- •History of severe impairment of the immunological system
- •Chronic conditions representing a contraindication to the use of steroid drugs
- •Non compliance with the study request
- •Any previous steroid assumption within 30 days before starting ERY-DEX
- •Have any other significant disease that in the Investigator's opinion would exclude the patient from the trial
- •Females of childbearing potential who were pregnant, breast-feeding or were not using adequate contraceptive methods
研究组 & 干预措施
ERY-DEX
Patients treated with monthly treatment of ERY-DEX (dexamethasone sodium phosphate encapsulated in autologous erythrocytes)
干预措施: Dexamethasone (Drug)
结局指标
主要结局
Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - ITT Population
时间窗: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)
ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.
Mean Change From Baseline in Neurological Symptoms Assessed by Using International Cooperative Ataxia Rating Scale (ICARS) Score - PP Population
时间窗: At visits 2 (day 30), 4 (day 90) and 7 (final visit, day 180)
ICARS ia a 100-point semiquantitative scale, which goes from 0 to 100, offering a compartimentalised quantification of 4 subscores: Posture and Gait Disturbances (maximum score = 34), Kinetic Functions (maximum score = 52), Speech Disorders (maximum score = 8), and Oculomotor Disorders (maximum score = 6) for a possible total score of 100 points. The minimum score is 0 (normal), while the maximum score is 100 and corresponds to the worst status of the patient. This means that for each subscale, higher scores indicate higher levels of impairment.
次要结局
- Change From V4 to V7 in Investigator Global Assessment (IGA) - ITT Population(At visit 7 (final visit, day 180))
- Change From V4 to V7 in Investigator Global Assessment (IGA) - PP Population(At visit 7 (final visit, day 180))
- Mean Change From V4 to V7 in Ocular Motility - ITT Population(At visit 7 (final visit, day 180))
- Mean Change From V4 to V7 in Ocular Motility - PP Population(At visit 7 (final visit, day 180))
- Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - ITT Population(At visits 4 (90 days) and 7 (180 days, final visit))
- Change From Baseline for Vineland Adaptive Behaviour Scale (VABS) Total Score - PP Population(At visits 4 (90 days) and 7 (180 days, final visit))
- Count of Participants on ERY-DEX Showing Treatment Emergent Adverse Events (TEAEs) Including Serious Adverse Events (SAEs)(Throughout the study, until the end of 6 months of treatment (day 180))