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

NCT07518810 | Not Yet Recruiting Phase 2 DMC

• 1 other identifier: 2025-1012
• Study Type: interventional
• Target: 150
• Locations: 1 country
• Sites: 6

Brief Summary

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. 1.To evaluate whether NBP soft capsules, compared with placebo, alleviates the major clinical symptoms in patients with MSA.
2. 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
3. 3.Take NBP or Placebo three times a day for 6 months
4. 4.Be served with clinical visit four times for follow-up and tests
5. 5.Keep a diary of drug application and symptom changes

Conditions

Multiple System Atrophy

Keywords

multiple system atrophy; 3-n-butylphthalide

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (8)

• Overall symptom control of MSA

  Baseline; 1st, 3rd, 6th, 12th month after intervention initiation

• Autonomic function of MSA

  Baseline; 6th, 12th month after intervention initiation

• Depressive symptom of MSA

  Baseline; 6th, 12th month after intervention initiation

• Cognitive function of MSA

  Baseline; 6th, 12th month after intervention initiation

• Life quality of MSA

  Baseline; 6th, 12th month after intervention initiation

Other Outcomes (2)

• α-Syn aggregates

  Baseline; 6th month after intervention initiation

• Plasma Neuro-filament light chain

  Baseline; 6th month after intervention initiation

Study Arms (2)

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.

Drug: 3-N-butylphthalide

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.

Drug: 3-N-butylphthalide

Interventions

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.

NBP treatment group; Placebo control group

Eligibility Criteria

• Age: 30 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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.

Sponsors & Collaborators

• The Second Affiliated Hospital of Jiaxing University: collaborator
• Beijing Chao Yang Hospital: collaborator
• Huzhou Central Hospital: collaborator
• First Affiliated Hospital of Guangxi Medical University: collaborator
• Ningbo No.2 Hospital: collaborator
• Second Affiliated Hospital, School of Medicine, Zhejiang University: lead
• Taizhou Hospital of Zhejiang Province affiliated to Wenzhou Medical University: collaborator

Study Sites (6)

The First Affiliated Hospital of Guangxi Medical University

Nanning, Guangxi, China

Location

HuZhou Central Hospital

Huzhou, Zhejiang, China

Location

The Second Hospital of Jiaxing

Jiaxing, Zhejiang, China

Location

Ningbo Second Hospital

Ningbo, Zhejiang, China

Location

Taizhou Hospital of Zhejiang Province

Taizhou, Zhejiang, China

Location

Affiliated Beijing Chaoyang Hospital of Capital Medical University

Beijing, China

Location

MeSH Terms

Conditions

Multiple System Atrophy

Interventions

3-n-butylphthalide

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Jiali Pu, MD

  2nd Affiliated Hospital, School of Medicine, Zhejiang University, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jiali Pu, MD

CONTACT

+86 13989468062; jialipu@zju.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: Participants are assigned to NBP group or Placebo group in parallel for the duration of the study

Study Record Dates

• First Submitted: March 26, 2026
• First Posted: April 9, 2026
• Study Start: April 10, 2026
• Primary Completion (Estimated): April 10, 2027
• Study Completion (Estimated): October 10, 2027
• Last Updated: April 9, 2026

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: 1 year after trial completion

Locations

CN (6)