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

Official title

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

Overview

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.

Interventions

• 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.
• 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.

Primary outcome measures

• Change from Baseline in Total "ON" Time without Troublesome Dyskinesia as assessed by 24-hour patient diary cards. [Time frame: Baseline, Day 85]

Eligibility criteria

Inclusion criteria

• Age ≥ 30 years at screening.
• Clinical diagnosis of clinically established or clinically probable Parkinson's disease.
• 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.).
• Hoehn-Yahr stage ≥2 in the "OFF" state.

Exclusion criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

Randomized

Model

Parallel assignment

Masking

Quadruple blind

Primary purpose

Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07585565 · GM1-PD-201

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗