ClinicalTrials.gov record NCT07476352

Expansion Study of ALT001 in Patients With Multiple System Atrophy

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 7, 2026

Expansion Study of ALT001 in Patients With Multiple System Atrophy

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhaseEarly phase 1A very early study, usually in a small number of people, to learn how the body handles a treatment. It is not designed to show whether the treatment works.
Age30 years old to 75 years old
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

An open-label, single-arm study testing ALT001 given by intravenous infusion in people with clinically probable or established multiple system atrophy aged 30–75. All participants receive three 30-day treatment cycles with ALT001 given daily on the first 14 days of each cycle; safety and symptom measures are collected during treatment and up to about 180 days. The study enrolls about 60 participants and records adverse events and clinical scales such as UMSARS and COMPASS.

What it is studying. The study is testing the safety, tolerability, and preliminary clinical and biomarker effects of intravenous ALT001 in people with multiple system atrophy.

What the researchers hope to learn. Scientific goals are to evaluate safety and tolerability of ALT001 in people with MSA, to gather preliminary data on clinical measures (UMSARS, COMPASS, orthostatic hypotension) and on plasma biomarkers (NFL, α‑syn, GFAP), and to inform further research.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This is an open-label, single-center, prospective, single-arm clinical study. The primary objective of this study is to evaluate the safety, tolerability, and preliminary efficacy of ALT001 in the treatment of patients with multiple system atrophy (MSA) in a real-world setting.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • ALT001

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Age between 30 and 75 years inclusive, either sex;
  • Clinically established or clinically probable MSA (including both MSA-C and MSA-P subtypes);
  • Ability to walk independently or with the aid of a walking device for at least 10 meters;
  • Provision of written informed consent.

Exclusion criteria

  • Evidence of other central nervous system pathologies on brain MRI at screening suggesting a diagnosis of neurodegenerative diseases other than MSA;
  • Other significant pathological findings on brain MRI at screening, including but not limited to: cerebral hemorrhage, acute cerebral infarction, aneurysm, vascular malformation, infectious lesions, brain tumors, or other space-occupying lesions (meningiomas or arachnoid cysts with a maximum diameter <1 cm do not require exclusion);
  • Presence of immune-mediated diseases that are inadequately controlled or require treatment with biologic agents;
  • Known history of allergies to biologic agents, such as proteins or cell-based products;
  • Receipt of any vaccination within the past 1 month;
  • Patients with a prior definitive diagnosis of malignancy or those currently receiving anti-tumor drug therapy;
  • Patients with a prior definitive diagnosis of epilepsy or those currently taking anti-epileptic drugs;
  • Concurrent severe hepatic insufficiency, renal insufficiency, or severe cardiac insufficiency (severe hepatic insufficiency defined as ALT ≥1.5 times the upper limit of normal or AST ≥1.5 times the upper limit of normal; severe renal insufficiency defined as serum creatinine [CRE] ≥1.5 times the upper limit of normal or estimated glomerular filtration rate [eGFR] <40 mL/min/1.73 m²; severe cardiac insufficiency defined as NYHA class 3-4), or any significant abnormalities on physical examination, vital signs, laboratory tests, or electrocardiogram that, in the investigator's opinion, require further examination or treatment, or may interfere with the study procedures or safety;
  • Patients with a history of alcohol or substance abuse, or alcohol or substance dependence within the past 2 years;
  • Patients diagnosed with psychiatric disorders according to DSM-V criteria, or those with obvious suicidal intent;
  • Patients who are pregnant, lactating, or have the potential to become pregnant, or those planning a pregnancy;
  • Inability to comply with follow-up assessments due to other reasons;
  • Patients deemed by the investigator to be unsuitable for participation in this study.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Show the detailed description from the registry

Multiple system atrophy (MSA) is a progressive neurodegenerative disorder characterized by a blend of autonomic dysfunction, Parkinson's syndrome, and cerebellar syndrome. The incidence of MSA ranges from 1.9 to 4.9 per 100,000 individuals, with an average age of onset of 56.2 years. Among individuals aged 50 years and older, the prevalence stands at 3.0 per 100,000. The mean age of MSA onset is 56.2 years, and the median survival ranges from 6.2 to 7.5 years. MSA often presents with severe autonomic dysfunction early in its course, impacting patient survival. Furthermore, due to difficulties in early diagnosis, rapid progression, and poor prognosis, nearly 50% of patients require a walking aid or physical assistance for ambulation within three years of motor symptom onset. Within five years, 60% of patients become wheelchair-dependent, and after six to eight years, most are completely bedridden, severely compromising their quality of life. Current symptomatic and supportive therapies fall short of meeting the treatment requirements of MSA patients. Furthermore, potential adverse effects and disease progression factors restrict the use of certain drugs, highlighting the critical need for the development of disease-modifying or neuroprotective agents to decelerate disease advancement. "ALT001, a nerve repair protein created by Darwin Origin (Hubei) Biopharmaceutical Co.LTD, is derived from cellular exosomes, a set of specific microenvironmental protein polymers secreted by stem cells under emergency conditions. It boasts selective assembly, targeted delivery, highly efficient tissue repair, exceptional safety, chemical stability, and convenient storage. Previous basic research has indicated ALT001's potential for promoting endogenous neural tissue repair, exhibiting significant neuroprotective and neurorestorative effects in animal models. Therefore, building upon the previously initiated study of ALT001 in patients with the MSA-P subtype and the forthcoming study in patients with the MSA-C subtype, we are broadening the inclusion and exclusion criteria to evaluate the safety, tolerability, and efficacy of ALT001 in the treatment of MSA in a real-world setting. This study aims to recruit 60 MSA patients aged between 30 and 75 years. All participants will receive three cycles of ALT001 treatment, with each cycle lasting 30 days. ALT001 will be administered via intravenous infusion (130 μg) during the first 14 days of each cycle. Assessments including vital signs, laboratory tests (e.g., routine blood tests, blood biochemical examinations, coagulation tests), and neurological evaluations (e.g., Unified Multiple System Atrophy Rating Scale [UMSARS] and Composite Autonomic Symptom Score [COMPASS]) will be conducted at each cycle and at 180 days post-treatment. Additionally, if patients experience new neurological symptoms or suspicious events, additional visits will be carried out, and researchers are required to submit and interpret relevant data within 72 hours of the event occurrence.The protocol of this study has been approved by the Ethics Committee of Beijing Tiantan Hospital. All participants will provide written informed consents before entering the study.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants receive three 30‑day treatment cycles of ALT001 with daily intravenous infusions on days 1–14 of each cycle (130 μg per infusion, given in ~30–40 minutes). Clinic visits with vital signs, blood tests, and neurological evaluations including UMSARS and COMPASS occur at day 15, day 45 (±3), day 75 (±5), and at day 180 (±14); some biomarker and gait assessments are timed per the protocol. Extra visits will be arranged for new neurological symptoms or suspicious events; investigators must report and interpret relevant data within 72 hours and monitor adverse events through the ~180‑day period. The study record indicates ALT001 may be given by intravenous infusion (as described) or by intrathecal (spinal) injection, which is invasive — ask the study team which route applies to you. All participants must provide written informed consent before joining.

How the study is structured. Interventional, open-label (no blinding), single-group study. Everyone in the study receives ALT001 by IV infusion (130 μg dissolved in 100 ml sodium chloride) once daily on days 1–14 of three consecutive 30-day cycles. Clinical scales, autonomic testing, gait analysis, quality-of-life questionnaires, blood biomarker tests, and routine safety labs are collected at set visits during treatment and around 180 days after treatment; adverse events are monitored throughout.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on multiple system atrophy

Clinical trials76 trials listed for multiple system atrophyEvery trial the registry lists for multiple system atrophy, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in multiple system atrophy researchLatest monthly update: September 2026. Includes: Tube feeding. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Jun 15, 2026
Primary completion
Oct 30, 2027 (estimated)
Study completion
Oct 30, 2027 (estimated)
Target enrollment
60 participants (estimated)
Last updated on registry
Sep 9, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
yilong Wang

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • Beijing Tiantan HospitalRecruiting

    Beijing, Beijing Municipality, 100070 · China

Terms used on this page · explanations by MedicalUpdates.ai

open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
single-arm
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
primary objective
The main result the study is designed to measure and report on. Other results, called secondary outcomes, are also collected.
written informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
MRI
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
at screening
The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
estimated glomerular filtration rate
A blood-test estimate of how well the kidneys are filtering. Lower numbers mean reduced kidney function; many studies set a minimum value.
electrocardiogram
A recording of the heart's electrical activity. QTc is one measurement from it; some drugs are avoided if it is prolonged.
follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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