Multicentric, Randomized, Double-blind Study Versus Placebo, With Two Parallel Groups Treated to Evaluate the Efficacy and the Tolerance of Riluzole in Children and Young Adults (6 to 20 Years of Age) With SMA. (Type II and Type III)
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 141
- 试验地点
- 1
- 主要终点
- The drug could stabilize patients condition during 24 months, and especially interrupt paralysis progression: Motor function (MFM scale)
研究概览
简要总结
This is a multicentric, randomized, double-blind study versus placebo, with two parallel groups treated to evaluate the efficacy and the tolerance of Riluzole in children and young adults (6 to 20 years of age) with SMA. (Type II and Type III).
详细描述
ASIRI study should allow to evaluate the efficacy and the tolerance of Riluzole in children and young adults (6 to 20 years of age) with SMA. This is a multicentric, randomized, double-blind study versus placebo, with two parallel groups treated and followed during 2 years. It has been preceded by a 7 days pharmacokinetic phase, concerning 14 patients, aiming to provide information on the kinetic profile of Riluzole in children. The drug could stabilize patients condition, and especially interrupt paralysis progression; those are the desired effects.
There is an open-label study of the long term safety of riluzole therapy in spinal muscular atrophies types II and III, with patients previously enrolled in ASIRI double-blind study.
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 20 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients afflicted with spinal muscular atrophy, type II or III, with genetic defect confirmed.
- •Age between 6 and 20 years old.
- •Score MFM at least 12
- •Negative pregnancy test for women of child-bearing age
- •Signing of an informed consent form, after appropriate information has been provided (if the patient is under 18 years old, both parents are required to sign the form too; otherwise, only her (his) agreement is necessary).
排除标准
- •Patients already treated with Riluzole
- •Concomitant treatment with: GAPAPENTINE, DEXTROMETHORPHANE, amantadine, any hepatotoxic medication that cannot be stopped, any other experimental product
- •Hepatic insufficiency: SGPT and/or SGOT levels higher than or equal to twice the normal higher limit
- •Renal insufficiency (creatinine above 115 micromoles/l)
- •Severe cardiac insufficiency
- •Current pneumopathy (clinical signs of an acute episode, confirmed by pulmonary X-ray, requiring specific treatment)
- •Pregnancy or nursing for women; non-abstinence or absence of effective contraception for nubile women
- •Any pathology or other circumstance likely to interfere with a regular follow-up
- •No affiliation to any social insurance system
研究组 & 干预措施
Placebo
MAIN EXCIPIENT OF THE RILUTEK
干预措施: Riluzole (Drug)
Riluzole
RILUTEK
干预措施: Riluzole (Drug)
结局指标
主要结局
The drug could stabilize patients condition during 24 months, and especially interrupt paralysis progression: Motor function (MFM scale)
时间窗: 6, 12, 18 and 24 months
次要结局
- Forced vital capacity (spirometry)(6, 12, 18 and 24 months)
- Quality of life (OKado questionnaire)(12 and 24 months)
- Measure of functional independence (MFI)(6,12,18 and 24 months)
- Tolerance evaluation: Somatic symptoms and adverse events Blood pressure and heart rate Weight and size Blood count, hepatic enzymes and bilirubin(every 3 months)