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New protein therapy hopes to repair brain damage in rare disease

NCT ID NCT06765733

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 26, 2026 · Last updated Jun 26, 2026

Summary

This early-phase trial tests a drug called Aleeto, a nerve repair protein made from stem cells, in 20 people with Multiple System Atrophy (MSA), a rare brain disease. The study checks if Aleeto is safe and might help with symptoms. Participants receive the drug via injection into the spine or vein.

What this could mean

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

Active substance
Aleeto (a nerve repair protein derived from stem cells)
What this could lead to
If it works, this could point toward a treatment that repairs nerve damage and slows progression of Multiple System Atrophy.
What could go wrong
This is a very early, small trial (20 people) focused on safety, not proof of effectiveness. The treatment may not work or could cause side effects.

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 20 people

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

Started

May 2025

Expected to finish

Dec 2026

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: 1.30 years ≤ 75 years of age, regardless of sex; 2.Clinically confirmed or clinically probable MSA-P; 3.Poor response to levodopa; 4.MSA-related motor symptom onset ≤5 years at first visit; 5.Walking ≥10 meters independently or with a walking aid; 6.Expected survival of ≥1 year, as determined by the investigator; 7.Signed informed consent. Exclusion Criteria: 1. Head MRI at screening showing evidence of other CNS lesions consistent with a diagnosis of neurodegenerative disease other than MSA; 2. Patients with MMSE scores indicative of dementia prior to enrolment (≤17 points for illiterate individuals, ≤20 points for individuals with elementary school education, ≤24 points for individuals with junior high school education or higher) or those with a prior confirmed diagnosis of dementia; 3. Head MRI at screening showing other significant pathological findings including but not limited to: cerebral hemorrhage, acute phase of cerebral infarction, aneurysm, vascular malformation, infectious lesion, brain tumor or other space-occupying lesion (meningiomas or arachnoid cysts with a maximum diameter of \<1 cm need not be excluded); 4. Presence of immune disorders that are inadequately controlled or require treatment with biological agents; 5. Known history of allergy to biological agents such as proteins and cell products; 6. Patients who have received any vaccination within 1 month; 7. Patients with pre-existing, clearly diagnosed malignant tumor or being treated with anti-tumor drugs; 8. Patients with a history of clearly diagnosed epilepsy or taking antiepileptic drugs; 9. Presence of lumbar spine disease and deformity or other contraindications to lumbar puncture; 10. Patients with abnormal coagulation function prior to enrolment (e.g., platelet count \<100 × 10E9/L; prothrombin time \[PT\] \>3 s), previous diagnosis of coagulation disorders such as hemophilia, and patients currently receiving more than two types of antiplatelet medication; 11. Contraindications to MRI (e.g. claustrophobia, internal placement of pacemakers or paramagnetic metals, etc.); 12. With severe hepatic insufficiency, renal insufficiency or severe cardiac insufficiency (severe hepatic insufficiency refers to ALT value≥2.0 times the upper limit of normal value or AST value≥2.0 times the upper limit of normal value; severe renal insufficiency refers to CRE≥1.5 times the upper limit of normal value or eGFR\<40mL/min/1.73m2; severe cardiac insufficiency refers to NYHA class 3-4); 13. Hepatitis B active infection (hepatitis B surface antigen positive and/or serum HBV DNA positive or serum HBV DNA \> 2 × 10E8 IU/ml; 14. Hepatitis C virus antibody positive or history of positive test; 15. Positive HIV test or history of positive test; 16. Patients with a combined history of alcohol or drug abuse or alcohol or drug dependence within 2 years; 17. Other psychiatric disorders diagnosed according to DSM-V diagnostic criteria, or significant suicide intent; 18. Patients who are pregnant, breast-feeding, or who are likely to become pregnant and plan to become pregnant; 19. Patients who are participating in other interventional studies or using other investigational biological agents, drugs, or devices, and patients who have used other experimental drugs within 1 month or within 5 drug half-lives; 20. Unable to be cooperative and complete the follow-up due to other reasons. 21. Patients who, in the opinion of the investigator, are not suitable for participation in this trial.

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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, Capital Medical University

    RECRUITING

    Beijing, Beijing Municipality, 100070, China

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