Aleeto for Parkinsonian multiple system atrophy (MSA-P)

The Study of Safety and Preliminary Efficacy of Aleeto in Patients With MultIple System Atrophy

This study will test whether Aleeto is safe and might help people with the Parkinsonian form of multiple system atrophy.

Quick facts

• Phase: Early Phase 1
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 30 Years to 75 Years
• Sex: All
• Sponsor: Beijing Tiantan Hospital (Academic / other)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT06765733 on ClinicalTrials.gov

What this trial studies

This is a single-center, prospective, randomized, open-label study with blinded outcome assessment (PROBE) conducted at Beijing Tiantan Hospital to test safety, tolerability, and preliminary efficacy of Aleeto in patients with MSA-P. Aleeto is a nerve‑repair protein therapy developed by Darwin Origin (Beijing) Biopha that is being given to participants and monitored for adverse events and clinical changes. Eligible participants are adults with clinically probable or confirmed MSA-P, poor response to levodopa, symptom onset within five years, and the ability to walk at least 10 meters. Those who complete the core study may opt into an open-label extension for additional follow-up.

Who should consider this trial

Good fit: Adults aged 30–75 with clinically probable or confirmed MSA-P, poor levodopa response, motor symptom onset within 5 years, ability to walk ≥10 meters (with or without aid), expected survival ≥1 year, and who can give informed consent are ideal candidates.

Not a fit: People with dementia by MMSE criteria, other significant CNS pathology on MRI, disease onset beyond five years, or who cannot walk 10 meters are unlikely to benefit from this early-phase intervention.

Why it matters

Potential benefit: If successful, Aleeto could provide a safe neurorepair therapy that slows disease progression or improves motor and autonomic function in people with MSA-P.

How similar studies have performed: Approaches aiming at neurorepair or exosome-related therapies remain experimental with only limited early-phase human data and no proven disease-modifying treatment for MSA so far.

Eligibility criteria

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.

Where this trial is running

Beijing, Beijing Municipality

• Beijing Tiantan Hospital, Capital Medical University — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Principal investigator: Yilong Wang, M.D. — Beijing Tiantan Hospital, Capital Medical University, Beijing, China
• Study coordinator: Weiqi Chen, M.D.

Conditions Multiple System Atrophy - Parkinsonian Subtype; Multiple System Atrophy, MSA; Multiple System Atrophy; Treatment; Exosomes; Neurorepair