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Experimental protein therapy targets rare brain disease

NCT ID NCT07476352

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 10, 2026 · Updated 2 times

Summary

This early-phase trial tests a drug called ALT001, a nerve repair protein, in 60 people with multiple system atrophy (MSA), a rare and progressive brain disorder. The study is open-label, meaning everyone knows they are getting the drug, and it focuses on safety and whether the drug shows any signs of helping. Participants receive ALT001 through a spinal injection or IV.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
ALT001 (a nerve repair protein derived from stem cells)
What this could lead to
If it works, this could point toward a treatment that slows or repairs nerve damage in multiple system atrophy.
What could go wrong
This is a very early (Phase 1), small, open-label trial with no placebo group. Results may not confirm effectiveness, and safety is still being evaluated.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

About 60 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Oct 2027

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

30 to 75 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 1\. Age between 30 and 75 years inclusive, either sex; * 2\. Clinically established or clinically probable MSA (including both MSA-C and MSA-P subtypes); * 3\. Ability to walk independently or with the aid of a walking device for at least 10 meters; * 4\. Provision of written informed consent. Exclusion Criteria: * 1\. Evidence of other central nervous system pathologies on brain MRI at screening suggesting a diagnosis of neurodegenerative diseases other than MSA; * 2\. 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); * 3\. Presence of immune-mediated diseases that are inadequately controlled or require treatment with biologic agents; * 4\. Known history of allergies to biologic agents, such as proteins or cell-based products; * 5\. Receipt of any vaccination within the past 1 month; * 6\. Patients with a prior definitive diagnosis of malignancy or those currently receiving anti-tumor drug therapy; * 7\. Patients with a prior definitive diagnosis of epilepsy or those currently taking anti-epileptic drugs; * 8\. 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; * 9\. Patients with a history of alcohol or substance abuse, or alcohol or substance dependence within the past 2 years; * 10\. Patients diagnosed with psychiatric disorders according to DSM-V criteria, or those with obvious suicidal intent; * 11\. Patients who are pregnant, lactating, or have the potential to become pregnant, or those planning a pregnancy; * 12\. Inability to comply with follow-up assessments due to other reasons; * 13\. Patients deemed by the investigator to be unsuitable for participation in this study.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Beijing Tiantan Hospital

    RECRUITING

    Beijing, Beijing Municipality, 100070, China

More trials for these conditions

Other studies related to the condition(s) this trial covers.