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

Trial details

Who runs it

Beijing Tiantan Hospital

Trial code

NCT06765733

Record updated

3 September 2025

Original description

This is a single-center, prospective, randomized, open-label, blinded outcome assessment (PROBE) study. At the end of the PROBE study, patients who have completed the study may opt to enter the open-label extension (OLE) study. The objective of the study is to evaluate the safety, tolerability and potential preliminary efficacy of Aleeto in the treatment of patients with multiple system atrophy (MSA).Read full original descriptionShow less

This is a single-center, prospective, randomized, open-label, blinded outcome assessment (PROBE) study. At the end of the PROBE study, patients who have completed the study may opt to enter the open-label extension (OLE) study. The objective of the study is to evaluate the safety, tolerability and potential preliminary efficacy of Aleeto in the treatment of patients with multiple system atrophy (MSA).

Inclusion: 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: 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; Presence of immune disorders that are inadequately controlled or require treatment with biological agents; Patients who have received any vaccination within 1 month; Patients with pre-existing, clearly diagnosed malignant tumor or being treated with anti-tumor drugs; 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; Head MRI at screening showing evidence of other CNS lesions consistent with a diagnosis of neurodegenerative disease other than MSA; 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); Known history of allergy to biological agents such as proteins and cell products; Patients with a history of clearly diagnosed epilepsy or taking antiepileptic drugs; Presence of lumbar spine disease and deformity or other contraindications to lumbar puncture; Hepatitis C virus antibody positive or history of positive test; Positive HIV test or history of positive test; Patients with a combined history of alcohol or drug abuse or alcohol or drug dependence within 2 years; Other psychiatric disorders diagnosed according to DSM-V diagnostic criteria, or significant suicide intent; 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; 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); Hepatitis B active infection (hepatitis B surface antigen positive and/or serum HBV DNA positive or serum HBV DNA > 2 × 10E8 IU/ml; Contraindications to MRI (e.g. claustrophobia, internal placement of pacemakers or paramagnetic metals, etc.); Patients who are pregnant, breast-feeding, or who are likely to become pregnant and plan to become pregnant; Unable to be cooperative and complete the follow-up due to other reasons; Patients who, in the opinion of the investigator, are not suitable for participation in this trial.

ClinicalTrials.gov NCT06765733