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| Name | Class |
|---|---|
| The Second Affiliated Hospital of Jiaxing University | OTHER |
| Beijing Chao Yang Hospital | OTHER |
| Huzhou Central Hospital | OTHER |
| Taizhou Hospital of Zhejiang Province affiliated to Wenzhou Medical University |
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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:
Participants of ENMSA will:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NBP treatment group | Experimental | Participants in the NBP treatment group will receive NBP soft capsules (dosage form: 100 mg/capsule), two capsules per dose, three times daily (total daily dose of 600 mg). The whole treatment duration is six months, during which all patients will maintain their original medication unchanged. |
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| Placebo control group | Placebo Comparator | Participants in the Placebo control group will receive two Placebo soft capsules each time, three times daily (total daily dose of 600 mg). The whole application duration is six months, during which all patients will maintain their original medication unchanged. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| 3-N-butylphthalide | Drug | 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). 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Baseline; 1st, 3rd, 6th, 12th month after intervention initiation |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Baseline; 1st, 3rd, 6th, 12th month after intervention initiation |
| Measure | Description | Time Frame |
|---|---|---|
| α-Syn aggregates | Use blood sample collected from participents to evalute the α-Syn aggregates in blood | Baseline; 6th month after intervention initiation |
| Plasma Neuro-filament light chain | Use plasma sample collected from participents to evalute the NfL concentration in MSA patients |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Jiali Pu, MD | Contact | +86 13989468062 | jialipu@zju.edu.cn |
| Name | Affiliation | Role |
|---|---|---|
| Jiali Pu, MD | 2nd Affiliated Hospital, School of Medicine, Zhejiang University, China | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The First Affiliated Hospital of Guangxi Medical University | Nanning | Guangxi | China |
Share IPD within 1 year after trail completion by public study protocol, CRF,Email Contact, etc.
1 year after trial completion
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| ID | Term |
|---|---|
| D019578 | Multiple System Atrophy |
| ID | Term |
|---|---|
| D054969 | Primary Dysautonomias |
| D001342 | Autonomic Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D001480 | Basal Ganglia Diseases |
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| ID | Term |
|---|---|
| C027125 | 3-n-butylphthalide |
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| OTHER |
| First Affiliated Hospital of Guangxi Medical University | OTHER |
| Ningbo No.2 Hospital | OTHER |
Participants are assigned to NBP group or Placebo group in parallel for the duration of the study
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| 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 |
| Baseline; 6th, 12th month after intervention initiation |
| 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 | Baseline; 6th, 12th month after intervention initiation |
| 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 | Baseline; 6th, 12th month after intervention initiation |
| Life quality of MSA | Use Multiple System Atrophy-Quality of Life (MSA-QoL; score range: 0-160; higher score means worse life quality of MSA) scale to evaluate life quality of MSA patients | Baseline; 6th, 12th month after intervention initiation |
| Safety of Butylphthalide application in MSA patients | Monitoring the incidence of AE/SAE and MSA specific mortality rate during the trial | through study completion, an average of 1 year |
| Anxiety symptoms of MSA | Use Hamilton Anxiety scale (HAMA; score range: 0-56; higher score means worse anxiety symptoms of MSA) to evaluate anxiety symptoms of MSA | Baseline; 6th, 12th month after intervention initiation |
| Advanced Cognitive function of MSA | Use Montreal Cognitive Assessment (MoCA; score range: 0-30; higher score means better cognitive function of MSA) to evaluate cognitive symptoms of MSA | Baseline; 6th, 12th month after intervention initiation |
| Baseline; 6th month after intervention initiation |
| HuZhou Central Hospital | Huzhou | Zhejiang | China |
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| The Second Hospital of Jiaxing | Jiaxing | Zhejiang | China |
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| Ningbo Second Hospital | Ningbo | Zhejiang | China |
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| Taizhou Hospital of Zhejiang Province | Taizhou | Zhejiang | China |
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| Affiliated Beijing Chaoyang Hospital of Capital Medical University | Beijing | China |
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| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009069 | Movement Disorders |
| D000080874 | Synucleinopathies |
| D019636 | Neurodegenerative Diseases |