Efficacy, Safety and Tolerability of Growth Hormone in Patients With Amyotrophic Lateral Sclerosis as add-on Therapy to Riluzole
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 40
- 试验地点
- 2
- 主要终点
- Primary endpoint is the N-acetylaspartate/Creatine ratio in the motor cortex assessed with magnetic resonance spectroscopy.
研究概览
简要总结
Several drugs have been proposed for ALS. These drugs included: Topiramate, Lamotrigine, creatine, Vit. E, Pentoxifylline, etc. Although most of the trials showed a positive trend, none of them reached a statistically significant result. The only exception is the Riluzole trial, that demonstrated a small but significant reduction in mortality between treated and untreated patients. Aim of our study is to determine if the add-on of GH to treatment with Riluzole is able to reduce neuronal loss in the motor cortex of ALS patients.
详细描述
Several drugs have been proposed for ALS. These drugs included: Topiramate, Lamotrigine, creatine, Vit. E, Pentoxifylline. Although most of the trials showed a positive trend, none of them reached a statistically significant result. The only exception is the Riluzole trial, that demonstrated a small but significant reduction in mortality between treated and untreated patients. When administered to SOD-1 transgenic mice, IGF-I prolongs survival, ameliorates muscular strength, and reduces weight and motor neuron loss, astrocyte gliosis, and ubiquitin positive protein inclusions.
Two clinical trials have been performed in ALS patients with s.c. administration of IGF-I indicating a possible beneficial effect, and a third clinical trial is in progress. Methionyl growth hormone (mGH) showed no effect on survival, disease progression and muscular strength. MGH was administered at a fixed dose and peripheral production of IGF-I appeared to be normal. We propose a double-blind trial of Growth Hormone (GH) as add-on therapy to Riluzole, with an individually regulated dose based on the peripheral response of IGF-I. Aim of our study is to determine if the add-on of GH to treatment with Riluzole is able to reduce neuronal loss in the motor cortex of ALS patients. As secondary objectives, effect of GH on mortality, QoL, and motor function will be assessed.
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
- 40 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Definite/probable ALS according to El Escorial criteria
- •Aged > 40, < 85 years
- •Progression from onset
- •Disease duration ≤3 years
- •Treatment with Riluzole
排除标准
- •Rapid disease progression in the first 6 months after diagnosis
- •Patients with tracheostomy and/or Gastrostomy
- •Disease duration > 3 years
- •Patient with exclusive bulbar or 2° motorneuron involvement
- •Hepatic/renal failure
- •Pregnant or breastfeeding
- •Signs of active neoplasia
- •Complicated Diabetes
- •Severe hypertension
- •Unable to undergo MRI exams
研究组 & 干预措施
1
Patients randomly assigned to treatment
干预措施: Growth Hormone (Somatropin) (Drug)
2
Patients randomly assigned to placebo
干预措施: Placebo (Drug)
结局指标
主要结局
Primary endpoint is the N-acetylaspartate/Creatine ratio in the motor cortex assessed with magnetic resonance spectroscopy.
时间窗: 0, 6 and 12 months after treatment start
次要结局
- Difference in mortality between groups(12 months)
- Difference in the ALS-FRS score (motor function scale)(0, 6, and 12 months after treatment start)
- Difference in the SF-36 score (quality of life )(0, 6, and 12 monthst after treatmetn start)
- Safety and tolerability(12 months)