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

Trial tests if a drug called Butylphthalide improves symptoms in Multiple System Atrophy (MSA) patie

NCT: NCT07518810 · Status: NOT YET RECRUITING · Phase: Phase 3 · Sponsor: Second Affiliated Hospital, School of Medicine, Zhejiang University · Started: 2026-04-10 · Est. Completion: 2027-10-10

Plain English Summary

Efficacy and Safety of Butylphthalide in the Treatment of Multiple System Atrophy is a Phase 3 clinical trial sponsored by Second Affiliated Hospital, School of Medicine, Zhejiang University studying Multiple System Atrophy. This trial tests the drug Butylphthalide (NBP) against a placebo to see if it helps with symptoms of Multiple System Atrophy (MSA). It is for adults aged 30-80 diagnosed with MSA within the last 5 years, who can still cooperate with assessments. Participants will take NBP or a placebo three times daily for 6 months and attend 4 clinic visits for check-ups and tests. Currently, there are no approved treatments specifically for MSA, making this trial important for finding new options. The trial aims to enroll 150 participants.

Official 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. 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

Who Can Participate

Here is what you need to know about eligibility for this trial. You can join if you are between 30 and 80 years old, diagnosed with MSA within the last 5 years, and can still move around and cooperate with tests. You cannot join if you have other neurological conditions like Parkinson's disease, severe cognitive impairment, or a history of major brain issues. You also cannot join if you have severe liver or kidney problems, a recent bleeding event, or are pregnant, breastfeeding, or planning to become pregnant. This trial is studying Multiple System Atrophy, so participants generally need a confirmed diagnosis.

What They're Measuring

The main outcome measures how well the drug helps control the primary symptoms of MSA, indicating its potential to improve daily functioning and quality of life for patients. The specific primary outcome measures are: Main symptom control of MSA (Baseline; 1st, 3rd, 6th, 12th month after intervention initiation). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 3, the final and most rigorous stage before seeking FDA approval. Phase 3 trials involve 300-3,000+ patients across multiple sites and compare the new treatment directly against the current standard of care. These pivotal trials generate the evidence needed for regulatory review. About 58% of Phase 3 drugs receive FDA approval. Successful Phase 3 results typically lead to a New Drug Application submission.

Why This Trial Matters

This trial addresses a significant unmet need by investigating a potential new treatment to alleviate the debilitating symptoms of Multiple System Atrophy (MSA), a rare and progressive neurological di As a Phase 3 trial, positive results could directly lead to FDA approval, making this treatment available to the broader patient population. This research targets Multiple System Atrophy, where improved treatment options are needed.

Investor Insight

This Phase 3 trial for a drug already approved for stroke in China signals a potential expansion into a new therapeutic area, with a moderate market size due to MSA's rarity but significant unmet need Phase 3 trials have approximately a 50-60% chance of gaining FDA approval if they reach this stage.

Is This Trial Right for Me?

Ask your doctor if this trial is right for you, what the potential benefits and risks are, and how it might affect your current medications. Participation involves taking study medication daily for 6 months and attending 4 clinic visits for check-ups, tests, and symptom monitoring. You will need to keep a diary of your medication use and any changes in your symptoms. The trial is being conducted at 6 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

Interventions

Primary Outcomes

Secondary Outcomes

Full Eligibility Criteria

Inclusion Criteria:

1. 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;
2. 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;
3. 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;
4. 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

Exclusion Criteria:

1. 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.
2. Patients with a history of other major neurological disorders, including ischemic stroke, intracranial hemorrhage, epilepsy, encephalitis, or central nervous system demyelinating diseases;
3. Patients with a history of psychiatric disorders that may involve psychotic symptoms, such as schizophrenia, major depressive disorder, or dissociative -conversion disorders.
4. Patients with severe hepatic or renal impairment (alanine aminotransferase \[ALT\] or aspartate aminotransferase \[AST\] levels \>2 xULN; Estimated creatinine clearance \<30 mL/min;
5. Patients with a heamorrhage event within the past 3 months or a high bleeding risk;
6. Patients with a history of significant craniocerebral trauma or surgery;
7. Patients with severe cognitive impairment (Mini-Mental State Examination \[MMSE\] score \<24);
8. Patients with a history of malignancy or autoimmune diseases;
9. Patients with dysphagia due to severe medullary dysfunction or esophageal disorders, or those unable to comply with medication administration for other reasons;
10. Patients who are pregnant, lactating, or planning a pregnancy within the next year.

Trial Locations

Frequently Asked Questions

What is clinical trial NCT07518810?

NCT07518810 is a Phase 3 INTERVENTIONAL study titled "Efficacy and Safety of Butylphthalide in the Treatment of Multiple System Atrophy." It is currently not yet recruiting and is sponsored by Second Affiliated Hospital, School of Medicine, Zhejiang University. The trial targets enrollment of 150 participants.

What conditions does NCT07518810 study?

This trial investigates treatments for Multiple System Atrophy. The primary condition under study is Multiple System Atrophy.

What treatments are being tested in NCT07518810?

The interventions being studied include: 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). Fo

What does Phase 3 mean for NCT07518810?

Phase 3 trials are large-scale studies involving 300-3,000+ patients that compare the new treatment against existing standard treatments. Positive Phase 3 results are typically required for FDA approval.

What is the current status of NCT07518810?

This trial is currently "Not Yet Recruiting." It started on 2026-04-10. The estimated completion date is 2027-10-10.

Who is sponsoring NCT07518810?

NCT07518810 is sponsored by Second Affiliated Hospital, School of Medicine, Zhejiang University. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT07518810?

The trial aims to enroll 150 participants. The trial has not yet started recruiting.

How is NCT07518810 designed?

This is a interventional study, uses randomized allocation, follows a parallel design, employs quadruple masking. Masking means some participants and/or investigators do not know which treatment group a participant is in, which helps reduce bias.

What are the primary outcomes being measured in NCT07518810?

The primary outcome measures are: Main symptom control of MSA (Baseline; 1st, 3rd, 6th, 12th month after intervention initiation). These are the main endpoints researchers use to determine whether the treatment is effective.

Where is NCT07518810 being conducted?

This trial is being conducted at 6 sites, including Nanning, Guangxi; Huzhou, Zhejiang; Jiaxing, Zhejiang; Ningbo, Zhejiang and 2 more sites (China).

Where can I find official information about NCT07518810?

The official record for NCT07518810 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT07518810. This government database provides the most up-to-date and detailed information about the trial.

What is NCT07518810 testing in simple terms?

This trial tests the drug Butylphthalide (NBP) against a placebo to see if it helps with symptoms of Multiple System Atrophy (MSA). It is for adults aged 30-80 diagnosed with MSA within the last 5 years, who can still cooperate with assessments.

Why is this trial significant?

This trial addresses a significant unmet need by investigating a potential new treatment to alleviate the debilitating symptoms of Multiple System Atrophy (MSA), a rare and progressive neurological di As a Phase 3 trial, positive results could lead directly to regulatory approval and new treatment options for patients.

What are the potential risks of participating in NCT07518810?

The most common side effects of Butylphthalide in other studies include gastrointestinal issues like nausea or diarrhea, and potential headaches. As with any trial, there's a risk that the drug may not be effective or could cause unexpected side effects not yet identified. Participants should be aware of the possibility of bleeding events, especially if they have a history of them or are on blood-thinning medications. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT07518810?

Ask your doctor if this trial is right for you, what the potential benefits and risks are, and how it might affect your current medications. Participation involves taking study medication daily for 6 months and attending 4 clinic visits for check-ups, tests, and symptom monitoring. You will need to keep a diary of your medication use and any changes in your symptoms. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT07518810 signal from an investment perspective?

This Phase 3 trial for a drug already approved for stroke in China signals a potential expansion into a new therapeutic area, with a moderate market size due to MSA's rarity but significant unmet need This is a Phase 3 trial, which is the final pivotal stage before potential regulatory submission.

What happens if the treatment in this trial doesn't work?

Participants will take NBP or a placebo three times daily for 6 months and attend 4 clinic visits for check-ups and tests. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.

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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.