Multicenter, Randomized, Double Blind, Placebo Controlled Clinical Trial With Riluzole in Spinocerebellar Ataxia Type 2
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 42
- 试验地点
- 1
- 主要终点
- Change in Ataxia symptoms (Scale for the Assessment and Rating of Ataxia (SARA))
研究概览
简要总结
ATRIL is a multi-centric, double-blind randomized, two-arm controlled study. 42 SpinoCerebellar Ataxia type 2 (SCA2) patients, both gender, at least 18 years of age will be included.
Riluzole 50 mg will be administered (per os) twice a day, versus one group with placebo for 12 months.
Riluzole (Rilutek®) is a benzothiazole drug, market approved, for Amyotrophic Lateral Sclerosis (ALS). It delays the onset of ventilator-dependence or tracheostomy in selected patients and may increase survival.
Scale for the Assessment and Rating of Ataxia (SARA) will be used at M0, M6 and M12. To assess primary criterion, the percentage of patients with a decrease of at least 1 point of the SARA score between the inclusion visit, and Visit 3 (Months 12) will be calculated.
详细描述
Inherited cerebellar ataxias are genetically heterogeneous neurological disorders. They are characterized by ataxic gait and cerebellar dysarthria that progresses over time with loss of ambulation and speech. The mutations by expansions of CAG triplets in the genes ATXN1 (SCA1), ATXN 2 (SCA2), 3 (SCA3), CACNA1A (SCA6), ATXN 7 (SCA7), and TBP (SCA17) are responsible for 50% of hereditary forms There is no curative or preventive treatment. This phase III study is a multi-centric, double-blind randomized, two-arm controlled study (one group with 50 mg Riluzole twice a day versus one group with placebo), to measure the efficacy of treatment with riluzole in SCA2 patients during 12 months. Amelioration is defined by a 1 point decrease of the SARA score.
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
- Triple (Participant, Care Provider, Investigator)
盲法说明
Treatments will be presented in numbered boxes, labeled for this study according to the Good Manufacturing Practices by the General Agency of Equipment and Health Products (AGEPS).
Each numbered box will consist of 6 months of treatment: 20 blister packs of 20 active or placebo tablets
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetically diagnosed SCA2 (CAG triplet in ATXN2 ≥ 33)
- •At least 18 years of age
- •Signature of informed consent
- •Covered by social security
- •SARA score ≥ 5 and ≤ 26
- •Age at onset ≤ 50 years old
排除标准
- •Treated with riluzole prior to the study
- •Hepatotoxic medication
- •Hypersensitivity to the active substance or to any of the excipients
- •Serious systemic illnesses or conditions known for enhancing the side effects of riluzole
- •Contraindications for MRI examination
- •Participation in another therapeutic trial (3 months exclusion period)
- •Pregnancy or breastfeeding
- •Non abstinence or absence of effective contraception for women
- •Inability to understand information about the protocol
- •Persons deprived of their liberty by judicial or administrative decision
- •Adult subject under legal protection or unable to consent
- •Other ataxic syndromes than SCA2
研究组 & 干预措施
RILUZOLE
Riluzole PMCS 50 mg is presented as a round, biconvex, 8 mm diameter nearly white film-coated tablet. The tablets will be held under a blister of 20 tablets.
干预措施: Riluzole (Drug)
PLACEBO
The placebo PMCS 50 mg is presented as a round, biconvex, 8 mm diameter nearly white film-coated tablet matching the appearance of the Riluzole used in this study
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Ataxia symptoms (Scale for the Assessment and Rating of Ataxia (SARA))
时间窗: at 12 months.
To compare the proportion of patients with Scale for the Assessment and Rating of Ataxia (SARA) improvement (decrease) of at least one point from baseline to 12 months
次要结局
- Change in Ataxia symptoms (Composite Cerebellar Functional Severity (CCFS) score)(at 12 months)
- Change in extracerebellar symptoms (Inventory of Non-Ataxia Signs (INAS))(at 12 months)
- 12 months survival(at 12 months)