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

Официальное название

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

Обзор

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.

Вмешательства

• Препарат 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.
• Препарат 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.
• Препарат 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.
• Препарат 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.
• Препарат 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.
• Препарат 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.

Первичные конечные точки

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

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Рандомизированное

Модель

Параллельные группы

Маскирование

Четверное слепое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07585565 · GM1-PD-201

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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