New protein therapy hopes to repair brain damage in rare disease
NCT ID NCT06765733
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 Hide 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.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Multiple system atrophy (MSA) are added.
Genom att skicka in godkänner du våra Användarvillkor
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.
-
The places running it
1 site. The list below names each one and where it is.
-
The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
-
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
RECRUITINGBeijing, Beijing Municipality, 100070, China
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- New PET tracer aims to spot Parkinson's in the brain
- New brain scan tracer could spot Parkinson's and related disorder
- Can a multidisciplinary clinic improve life for MSA patients?
- New hope for rare brain disorder: drug trial targets MSA
- Hope for MSA: new drug aims to slow rare brain disease
- DNA hunt for rare brain disease genes begins