Insulin-like Growth Factor-1 in Amyotrophic Lateral Sclerosis (ALS)
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Mayo Clinic
- 入组人数
- 330
- 试验地点
- 20
- 主要终点
- Rate of Change in Composite Manual Muscle Testing (MMT) Score
研究概览
简要总结
The purpose of this multicenter study is to determine if insulin-like growth factor-1 (IGF-I) slows the progressive weakness in amyotrophic lateral sclerosis (ALS) patients. Study participants will be followed for 2 years once enrolled. They will receive either placebo or the active IGF-I. Examinations will take place at approximately 6-month intervals.
详细描述
The objective of this trial was to determine whether IGF-1 (MyotrophinTM) slows progression of weakness in amyotrophic lateral sclerosis (ALS). Three hundred thirty patients with ALS from 20 medical centers participated in this double blind, placebo-controlled two-year study. Half the patients received IGF-1 and the other half received placebo. The drug will be administered twice a day.
ALS is a neurodegenerative disorder that causes progressive muscle weakness and loss of motor neurons. IGF-1 is a neurotrophic factor essential for normal development of the nervous system and shows protection of motor neurons in animal models and cell culture systems. It is thought to block cell death pathways and promote muscle re-innervation and axonal growth and regeneration.
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
- 18 Years 至 80 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- •Patients entering this study will not:
- •Have any of the following conditions:renal disease (Creatine > 2.0) or other active systemic disease
- •Have any clinically significant abnormalities on the prestudy laboratory evaluation, physical examination, ECG, chest x-ray or ophthalmologic exam.
- •Have any clinically significant medical condition (e.g., within six months of baseline, had myocardial infarction, angina pectoris, and/or congestive heart failure) that, in the opinion of the investigator, would compromise the safety of patient.
- •Have Type I or Type II diabetes.
- •Have a history of cancer including melanoma with the exception of localized skin cancers (with no evidence of metastasis, significant invasion, or re-occurrence within three years of baseline) and carcinoma in-situ of the cervix (women only).
- •Have used an investigational drug within 30 days of baseline visit.
- •Have had a tracheostomy.
- •Have a Beck's Depression Inventory score *
- •Have legal residency outside of the United States or Canada.
- •Be pregnant or breast-feeding.
研究组 & 干预措施
IGF-1
Insulin like growth factor, type 1 will be given 0.05 mg per kg body weight subcutaneously twice daily
干预措施: Insulin like growth factor, type 1 (Drug)
Placebo
Placebo arm
干预措施: Placebo (Drug)
结局指标
主要结局
Rate of Change in Composite Manual Muscle Testing (MMT) Score
时间窗: Baseline and 24 months
The primary outcome measure was the rate of change in the MMT score. MMT involved the examination of 34 muscle groups with standard positioning. The final MMT score represented an average of the 34 muscles examined, and ranged from 10 to 0(10 normal strength, 0 paralyzed). The individual muscle score was based on the medical research council (MRC) grading scale (1-5) modified to a 10 point system corresponding to the MRC modifications of plus and minus (5, 5-,4+,4,4-,3+,3, 3-,2,1,0; with 5 being normal strength and 0 paralyzed).
次要结局
- Number of Participants Alive and Tracheostomy-free at 24 Months(baseline to 24 months)
- Rate of Change in ALS Functional Rating Scale.(Baseline and 24 months)