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
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
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;
Parallel
3-N-butylphthalide
Drug3-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). For NBP used in ENMSA trial, its dosage form is soft capsule, containing 100mg NBP per capsule. Application frequency will be three times a day, 2 capsules each time.
Main symptom control of MSA
Use sum score Movement Disorder Society-Unified Multiple System Atrophy Rating Scale(MDS-UMSARS; score range: 0-104; higher score means worse symptoms of MSA) part I+II to evaluate the effectiveness of main symptom control of MSA
Overall symptom control of MSA
Use sub score of Movement Disorder Society-Unified Multiple System Atrophy Rating Scale(MDS-UMSARS; score range: 0-104; higher score means worse symptoms of MSA) to evaluate the overall effectiveness of main symptom control of MSA
Autonomic function of MSA
Use Composite Autonomic Symptom Score 31 (COMPASS-31; score range: 0-100; higher score means worse autonomic symptoms of MSA) scale to evaluate autonomic symptoms of MSA
Depressive symptom of MSA
Use Hamilton Depression Scale (HAMD; score range: 0-52; higher score means worse depressive symptoms of MSA) scale to evaluate depressive symptom of MSA
Cognitive function of MSA
Use Mini-mental State Examination scale (MMSE; score range: 0-30; higher score means better cognitive function of MSA patients) to evaluate cognitive symptoms of MSA
α-Syn aggregates
Use blood sample collected from participents to evalute the α-Syn aggregates in blood
Plasma Neuro-filament light chain
Use plasma sample collected from participents to evalute the NfL concentration in MSA patients
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Second Affiliated Hospital, School of Medicine, Zhejiang University
Lead sponsor
The Second Affiliated Hospital of Jiaxing University
Collaborator
Beijing Chao Yang Hospital
Collaborator
Huzhou Central Hospital
Collaborator
Taizhou Hospital of Zhejiang Province affiliated to Wenzhou Medical University
Collaborator
First Affiliated Hospital of Guangxi Medical University
Collaborator
Ningbo No.2 Hospital
Collaborator
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