Feasibility and Safety Pilot Therapeutic Study of the Iron Chelator Deferiprone in Amyotrophic Lateral Sclerosis
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 23
- 试验地点
- 1
- 主要终点
- Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale
研究概览
简要总结
The FAIR-ALS study is to investigate the safety and efficacy of a scavenger treatment of iron deferiprone, which would reduce the brain iron to limit the development of amyotrophic lateral sclerosis.
It has been shown an excess of iron in the central nervous system carrying a sporadic ALS patients. Iron overload associated with a loss of motor neurons may explain the signs of the disease (atrophy).
The investigators discuss the hypothesis that reducing excess iron, the investigators can reduce the loss of neurons and thus the progression of signs of the disease.
详细描述
At the end of the study, it will propose to continue the usual quarterly patient follow up, as recommended by the French ALS centers.
Deferiprone can be administered as part of a compassionate use, for patients who want it and who do not have hypoxemia.
We therefore plan a treatment period compassionate relatively short and less than 3 years.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Supportive Care
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Amyotrophic lateral sclerosis defined in accordance to the El Escorial criteria (possible, probable or defined)
- •18 to 85 years old patient, male or female
- •Patient with social security cover
排除标准
- •Achieved respiratory defined by a FVC <70%
- •Evolution of more than 24 months
- •Demented subject
- •Severe malnutrition
- •Patients with treatment potentially at risk of agranulocytosis and neutropenia
- •Patients with a history of agranulocytosis or iatrogenic under haematological disease
- •Incapable of giving consent
- •Indication against MRI
- •Indication against lumbar puncture
- •Patient refused lumbar puncture
- •Hypersensitivity to iron chelators
- •Concomitant treatment with antacids containing aluminum
- •Presence of another serious illness to life-threatening or disabling cons to the use of the treatment mixture of oxygen and nitrous oxide equally
研究组 & 干预措施
Deferiprone
Deferiprone, 25 to 30 mg/kg per day, oral use
干预措施: Deferiprone (Drug)
结局指标
主要结局
Evolution of Amyotrophic Lateral Sclerosis Functional Rating Scale
时间窗: V3, V6, V9, V12, V15
次要结局
- Comparison of the progression of ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale ) score for 3 months without treatment (V0 to V3) in the period of the first three months under treatment (V3 to V6).(SCREENING, V0, V3, V6)
- The proportion of patients who become non-self-sufficient after 12 months with the appearance of a sub scores ALSFRS-R less than or equal to 2 on swallowing, cut food using utensils or walk.(SCREENING, V0, V3, V6, V9, V12, V15)
- Number of patients with anemia at 12 months defined by a hemoglobin / dL Hb <12 g(V3, V6, V9, V12, V15)
- Number of serious and non-serious adverse events(SCREENING, V0, V3, V6, V9, V12, V15)
- Progression of respiratory vital capacity tests(SCREENING, V0, V3, V6, V9, V12, V15)
- Values of R2 * in MRI(V3, V6, V19)
- Oxidative stress markers analyzed blindly in blood and cerebrospinal fluid(V3, V9)
- No alteration of energy metabolism in aerobic and anaerobic blood and cerebrospinal fluid(V3, V9)