A Study to Evaluate the Efficacy and Safety of Monosialotetrahexosylganglioside Sodium Injection in Parkinson's Disease Participants With Motor Fluctuations

A Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Design Phase 2 Clinical Study to Evaluate the Efficacy and Safety of Monosialotetrahexosylganglioside Sodium Injection in Parkinson's Disease Participants With Motor Fluctuations

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Parkinson's Disease
• Intervention / Treatment: Drug: GM1; Drug: GM1; Drug: GM1; Drug: Placebo; Drug: Placebo; Drug: Placebo

• Study Type: Interventional
• Enrollment (Estimated): 276
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Tao Feng; Phone Number: +86 13911125339; Email: happyft@sina.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Age ≥ 30 years at screening.
2. Clinical diagnosis of clinically established or clinically probable Parkinson's disease.
3. Currently receiving levodopa therapy (stable regimen for ≥4 weeks prior to randomization, with a levodopa equivalent daily dose ≥400 mg) and experiencing motor fluctuations (including wearing-off, on-off phenomena, etc.).
4. Hoehn-Yahr stage ≥2 in the "OFF" state.

Exclusion Criteria:

1. History or presence of other Parkinsonian syndromes or Parkinson-plus syndromes, or hereditary neurodegenerative disorders.
2. Diagnosis of Parkinson's disease dementia or severe cognitive impairment as judged by the investigator at screening.
3. Prior surgical treatment for Parkinson's disease or planned such surgery during the trial period.
4. History or presence of other neurological diseases.
5. History or presence of autoimmune diseases.
6. History or presence of any demyelinating diseases, including but not limited to acute inflammatory demyelinating polyneuropathy (Guillain-Barré syndrome).
7. History or presence of hereditary glycolipid metabolism disorders .
8. Known allergy to monosialotetrahexosylganglioside sodium or any excipient of the investigational product.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

6

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Cohort 1 GM1	Drug: GM1; 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.; Drug: GM1; 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.; Drug: GM1; 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 Comparator: Cohort 1 Placebo	Drug: Placebo; 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.; Drug: Placebo; 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.; Drug: Placebo; 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.
Experimental: Cohort 2 GM1	Drug: GM1; 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.; Drug: GM1; 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.; Drug: GM1; 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 Comparator: Cohort 2 Placebo	Drug: Placebo; 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.; Drug: Placebo; 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.; Drug: Placebo; 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.
Experimental: Cohort 3 GM1	Drug: GM1; 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.; Drug: GM1; 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.; Drug: GM1; 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 Comparator: Cohort 3 Placebo	Drug: Placebo; 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.; Drug: Placebo; 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.; Drug: Placebo; 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Change from Baseline in Total "ON" Time without Troublesome Dyskinesia as assessed by 24-hour patient diary cards.	Baseline, Day 85

Collaborators and Investigators

• Sponsor: Qilu Pharmaceutical Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2026
• Primary Completion (Estimated): March 1, 2028
• Study Completion (Estimated): August 1, 2028

Study Registration Dates

• First Submitted: April 27, 2026
• First Submitted That Met QC Criteria: May 9, 2026
• First Posted (Actual): May 13, 2026

Study Record Updates

• Last Update Posted (Actual): May 13, 2026
• Last Update Submitted That Met QC Criteria: May 9, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Synucleinopathies; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurodegenerative Diseases; Movement Disorders; Parkinsonian Disorders; Basal Ganglia Diseases; Parkinson Disease
• Other Study ID Numbers: GM1-PD-201

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No