The Study of GM1 Ganglioside, A Potential New Parkinson's Disease Medication
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 94
- 试验地点
- 1
- 主要终点
- Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 24 Assessed Off Medication.
研究概览
简要总结
The purpose of this trial is to examine the short term effects (24 Weeks) of GM1 on Parkinson's disease (PD) symptoms, as well as the effects of long-term treatment (120 Weeks) with GM1 on disease progression, and to examine the extent to which GM1 treatment influences the underlying disease process in PD.
详细描述
The study is designed to further examine the extent to which GM1 ganglioside can improve symptoms, delay disease progression, and, perhaps, partially restore damaged brain cells in PD patients. GM1 ganglioside is a chemical that is normally found in the brain and is a normal part of the outer covering or membrane of nerve cells. This study will compare the effectiveness of GM1 to standard PD treatment. In addition to studying clinical measures of motor and cognitive functioning, the investigators will use PET (positron emission tomography) scanning to image the brain and the dopamine nerve endings in a subgroup of patients. Patients with mild to moderate idiopathic PD will be divided into 2 groups. One group will receive GM1 for 24 weeks and the other will receive placebo. At the end of this 24 week period, all patients will enter into a 96 week treatment period in which all patients will receive GM1.
In parallel, a group of standard-of-care patients with mild to moderate PD will be monitored over a 1 to 2 year period to assess the natural progression of PD. These patients will receive the same clinical evaluations as the treatment group subjects but they will not receive the experimental medication.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Crossover
- 主要目的
- Treatment
- 盲法
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 39 Years 至 85 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
- 未提供
研究组 & 干预措施
Early-Start Group
Subjects were randomized to receive GM1 ganglioside for 24 weeks.
干预措施: GM1 ganglioside (Drug)
Delayed-Start Group
Subjects were randomized to receive placebo for 24 weeks.
干预措施: Placebo (Drug)
结局指标
主要结局
Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 24 Assessed Off Medication.
时间窗: Baseline to Week 24
The Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) motor scores assess 14 symptoms some of which separately assess symptoms in different body parts (e.g. right arm, left arm, right leg, left leg) and each symptom is rated on a scale from 0 (normal) to 4 (severe). The minimum total score possible is 0 and the maximum total score possible is 108. Each subject was independently rated by two observers at each study visit and a mean score was calculated for analysis.
Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Scores From Baseline to Week 120 Assessed Off Medication.
时间窗: Baseline to Week 120
The Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) motor scores assess 14 symptoms some of which separately assess symptoms in different body parts (e.g. right arm, left arm, right leg, left leg) and each symptom is rated on a scale from 0 (normal) to 4 (severe). The minimum total score possible is 0 and the maximum total score possible is 108. Each subject was independently rated by two observers at each study visit and a mean score was calculated for analysis.
次要结局
- Change From Baseline to Week 24 in Total Unified Parkinson's Disease Rating Scale (UPDRS)Score Assessed Off Medication(Baseline to Week 24)
- Change in Total UPDRS Score From Baseline to Week 120 Assessed Off Medication(Baseline to Week 120)
- Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 24 Assessed Off Medication.(Baseline to Week 24)
- Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 48 Assessed Off Medication.(Baseline to Week 48)
- Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 72 Assessed Off Medication.(Baseline to Week 72)
- Change in Unified Parkinson's Disease Rating Scale (UPDRS) (Part III) Motor Score From Baseline to Week 96 Assessed Off Medication.(Baseline to Week 96)