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This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, phase 2 study. A total of 276 eligible Parkinson's disease participants with motor fluctuations will be enrolled and assigned to one of three cohorts. Within each cohort, participants will be randomized in a 3:1 ratio to receive either GM1 or matching placebo, resulting in six groups: Cohort 1 GM1 (n=69), Cohort 1 Placebo (n=23); Cohort 2 GM1 (n=69), Cohort 2 Placebo (n=23); Cohort 3 GM1 (n=69), Cohort 3 Placebo (n=23). All participants will continue their pre-enrollment anti-Parkinson's medication regimen as background therapy, which should remain as stable as possible during the study. The study consists of a 28-day screening period and an 85-day double-blind treatment period.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 GM1 | Experimental |
| |
| Cohort 1 Placebo | Placebo Comparator |
| |
| Cohort 2 GM1 | Experimental |
| |
| Cohort 2 Placebo | Placebo Comparator |
| |
| Cohort 3 GM1 | Experimental |
| |
| Cohort 3 Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| GM1 | Drug | 1000 mg on Day 1, followed by 400 mg once daily from Day 2 to the end of Week 2, then a 2-week drug holiday. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Change from Baseline in Total "ON" Time without Troublesome Dyskinesia as assessed by 24-hour patient diary cards. | Baseline, Day 85 |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Tao Feng | Contact | +86 13911125339 | happyft@sina.com |
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| GM1 | Drug | 1000 mg on Day 1, followed by 200 mg once daily from Day 2 to the end of Week 2, then a 2-week drug holiday. |
|
| GM1 | Drug | 1000 mg on Day 1, followed by 200 mg once daily from Day 2 to the end of Week 2. From Week 3 onward, 1000 mg once weekly until the last dose on Day 78. |
|
| Placebo | Drug | 1000 mg on Day 1, followed by 400 mg once daily from Day 2 to the end of Week 2, then a 2-week drug holiday. |
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| Placebo | Drug | 1000 mg on Day 1, followed by 200 mg once daily from Day 2 to the end of Week 2, then a 2-week drug holiday. |
|
| Placebo | Drug | 1000 mg on Day 1, followed by 200 mg once daily from Day 2 to the end of Week 2. From Week 3 onward, 1000 mg once weekly until the last dose on Day 78. |
|
| ID | Term |
|---|---|
| D010300 | Parkinson Disease |
| ID | Term |
|---|---|
| D020734 | Parkinsonian Disorders |
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |
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