Efficacy and Safety of Butylphthalide in the Treatment of Multiple System Atrophy

Официальное название

Efficacy and Safety of Butylphthalide in the Treatment of Multiple System Atrophy（ENMSA）: A Multicenter, Randomised, Double-blinded, Placebo-controlled Trial

Обзор

The present study aims to conduct a randomized controlled trial to evaluate the efficacy and safety of 3-n-Butylphthalide (NBP) in improving symptoms in patients with Multiple System Atrophy (MSA). The main questions it aims to answer are: 1. To evaluate whether NBP soft capsules, compared with placebo, alleviates the major clinical symptoms in patients with MSA. 2. Whether NBP application is safe to treat patients with MSA. In this trial, NBP will be compared with placebo (similar soft capsule without effective component of NBP) to demonstrate if NBP can alleviates MSA symptoms Participants of ENMSA will: 1. Take NBP or Placebo three times a day for 6 months 2. Be served with clinical visit four times for follow-up and tests 3. Keep a diary of drug application and symptom changes

Вмешательства

• Препарат 3-N-butylphthalide
  3-n-Butylphthalide (NBP), also known as celery seed oil extract, is a lipid-soluble compound isolated from celery seeds. NBP was approved by the China Food and Drug Administration (CFDA) in 2002 for the treatment of acute ischemic stroke. NBP has demonstrated significant improvement in motor deficits and exhibited neuroprotective effects in animal models of various neurodegenerative diseases, such as Alzheimer's disease (AD), Parkinson's disease (PD), and amyotrophic lateral sclerosis (ALS). Fo

Первичные конечные точки

• Main symptom control of MSA [Срок оценки: Baseline; 1st, 3rd, 6th, 12th month after intervention initiation]

Вторичные конечные точки (8)

• Overall symptom control of MSA [Срок оценки: Baseline; 1st, 3rd, 6th, 12th month after intervention initiation]
• Autonomic function of MSA [Срок оценки: Baseline; 6th, 12th month after intervention initiation]
• Depressive symptom of MSA [Срок оценки: Baseline; 6th, 12th month after intervention initiation]
• Cognitive function of MSA [Срок оценки: Baseline; 6th, 12th month after intervention initiation]
• Life quality of MSA [Срок оценки: Baseline; 6th, 12th month after intervention initiation]
• Safety of Butylphthalide application in MSA patients [Срок оценки: through study completion, an average of 1 year]
• Anxiety symptoms of MSA [Срок оценки: Baseline; 6th, 12th month after intervention initiation]
• Advanced Cognitive function of MSA [Срок оценки: Baseline; 6th, 12th month after intervention initiation]

Критерии участия

Критерии включения

• Meet a diagnosis for "clinically established MSA" according to the Movement Disorder Society (MDS) diagnostic criteria for multiple system atrophy revised in 2022, as assessed by a neurologist;
• Patients aged between 30 and 80 years, within 5 years since the initial diagnosis of MSA, and with a life expectancy greater than 3 years;
• Patients are not entirely dependent on a wheelchair or bedridden and are capable of cooperating with necessary assessments and examinations, including scale evaluations, magnetic resonance imaging (MRI), and PET-CT scans;
• Patients must have been on a stable medication regimen (for a duration of at least one month) prior to the trial, which may include drugs for anti-Parkinson, anti-autonomic dysfunction, anti-anxiety/depression agents, and sleep aids

Критерии исключения

• Patients with a diagnosis confirmed by PET-CT or revised during follow-up to other diseases, such as idiopathic Parkinson's disease, progressive supranuclear palsy, corticobasal degeneration, dementia with Lewy bodies, or secondary parkinsonian syndromes.
• Patients with a history of other major neurological disorders, including ischemic stroke, intracranial hemorrhage, epilepsy, encephalitis, or central nervous system demyelinating diseases;
• Patients with a history of psychiatric disorders that may involve psychotic symptoms, such as schizophrenia, major depressive disorder, or dissociative -conversion disorders.
• Patients with severe hepatic or renal impairment (alanine aminotransferase \[ALT\] or aspartate aminotransferase \[AST\] levels >2 xULN; Estimated creatinine clearance <30 mL/min;
• Patients with a heamorrhage event within the past 3 months or a high bleeding risk;
• Patients with a history of significant craniocerebral trauma or surgery;
• Patients with severe cognitive impairment (Mini-Mental State Examination \[MMSE\] score <24);
• Patients with a history of malignancy or autoimmune diseases;
• Patients with dysphagia due to severe medullary dysfunction or esophageal disorders, or those unable to comply with medication administration for other reasons;
• Patients who are pregnant, lactating, or planning a pregnancy within the next year.

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

Китай · 6 центров

• The First Affiliated Hospital of Guangxi Medical University — Nanning
• HuZhou Central Hospital — Huzhou
• The Second Hospital of Jiaxing — Jiaxing
• Ningbo Second Hospital — Ningbo
• Taizhou Hospital of Zhejiang Province — Taizhou
• Affiliated Beijing Chaoyang Hospital of Capital Medical University — Пекин

Идентификаторы

NCT: NCT07518810 · 2025-1012

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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