The Study of Safety and Preliminary Efficacy of ALT001 in Patients With MultIple System Atrophy-Cerebellar Type
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age30-75
Sponsoryilong Wang
About this trial
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 ALT001 in the treatment of patients with multiple system atrophy-cerebellar type (MSA-C).
Eligibility criteria
Qualifiers
1. Age between 30 and 75 years inclusive, either sex;
2. Clinically established or clinically probable MSA-C as defined by the 2022 MDS diagnostic criteria for multiple system atrophy;
3. Duration of MSA-C-related motor symptoms, including ataxia, no longer than 5 years;
4. Score ≤ 3 on Item 14 of the Unified Multiple System Atrophy Rating Scale (UMSARS) Part II (motor examination);
Disqualifiers
1. Presence of other diseases that may cause ataxia at screening (e.g., infectious diseases, immune-mediated diseases, tumors, paraneoplastic conditions, hereditary disorders, trauma, nutritional deficiencies, toxic exposure, or other neurodegenerative diseases);
2. Dementia indicated by the Mini-Mental State Examination (MMSE) at enrollment (score ≤17 for illiterate individuals, ≤20 for primary school education, or ≤24 for junior high school or above) or a prior definitive diagnosis of dementia;
3. Evidence of other central nervous system pathologies on brain MRI at screening suggesting a diagnosis of neurodegenerative diseases other than MSA; or 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);
4. Presence of immune-mediated diseases that are inadequately controlled or require treatment with biologic agents;
Trial design
Treatments tested in this trial
- ALT001
Treatment groups
20 Participants
are divided into 2 treatment groupsSponsors and collaborators
yilong Wang
Lead sponsor
Beijing Tiantan Hospital
Sponsor institution
Source ClinicalTrials.gov