Evaluation of the Efficiency of Treatment by BUMETANIDE on Autistic Children With a Known Ethiology : Multicenter and Double Blind-study With Randomized Parallel Group, Against Placebo.
试验速览
- 阶段
- 2 期
- 状态
- 撤回
- 发起方
- 试验地点
- 1
- 主要终点
- Change (evolution) between day 0 and day 99 of the result of the scale CARS (Childhood Autism Rating Scale).
研究概览
简要总结
During a previous therapeutic trial, investigators showed that the bumetanide improved significantly autism. This trial showed that a therapeutic response was obtained in 75% of cases.
These first results were reinforced by a study led with adult patients for whom the eye tracking measurements as well as the functional MRI showed a diminution of the response time and a modification (amplification) of the cerebral response during an emotions recognition test.
Finally, investigators confirmed the physiological mechanism behind the action of the bumetanide in a study in two mouse models of autism.
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)
入排标准
- 年龄范围
- 5 Years 至 17 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children and teenager from age 5 to age 17, with a diagnosis of typical autism or Asperger syndrome according to the criteria of diagnosis of the WHO's classification (CIM-10),
- •With a known etiology,
- •Patients for whom the CARS results are strictly Superior or equal to 30,
- •Of whom the parents have given their free, informed and written consent,
- •Affiliated or beneficiary of the French social security.
排除标准
- •Patients under treatment by inlet diuretic either at the time of the study or before,
- •Patients with electrolytic disorders,
- •Patients with a known hypersensitivity to sulfa drugs,
- •Patients with a hepatic or renal failure,
- •Patients with an epilepsy not controlled by a treatment (comitial crisis in the past 6 month at the time the trial starts despite a treatment),
- •Patients under treatment by psychotropic exception made of the melatonin,
- •Allergy to the bumetanide or one of its excipients,
- •Patient under a treatment by lithium, diphemanil, erythromycin IV, halofantrine, pentamidine, sultopride, vincamine, aminoglycoside,
- •Pregnant and lactating women.
- •Secondary exclusion criteria:
- •QT prolongation noticed on the ECG at Day0,
- •Anomaly on the biological check up (Day 0) made before including the patient that would contraindicated the prescription of bumetanide,
- •Patients for whom the CARS results are strictly inferior to 30.
研究组 & 干预措施
Placebo group
During 3 months in the double blind, the patient will receive the placebo. For the patient of 25kg and more the bumetanide is used at the posology of 1mg in the morning and 1mg in the evening, for patient under 25kg the posology is 0.5mg in the morning and 0.5mg in the evening.
After the 3 months in the double blind trial (bumetanide versus placebo), all the patient will receive (in the open phase of the trial) the bumetanide during 3 months with the posology fitting with their weights.
干预措施: Placebo (Drug)
Bumetanide group
During 3 months in the double blind, the patient will receive the experimental treatment. For the patient of 25kg and more the bumetanide is used at the posology of 1mg in the morning and 1mg in the evening, for patient under 25kg the posology is 0.5mg in the morning and 0.5mg in the evening.
After the 3 months in the double blind trial (bumetanide versus placebo), all the patient will receive (in the open phase of the trial) the bumetanide during 3 months with the posology fitting with their weights.
干预措施: Bumetanide (Drug)
结局指标
主要结局
Change (evolution) between day 0 and day 99 of the result of the scale CARS (Childhood Autism Rating Scale).
时间窗: Day 0 and Day 99
次要结局
- CARS (Childhood Autism Rating Scale) between D0 and D99 and between D99 and D190 which will be describe by etiology(Day 0, Day 99 and Day 190)