An Early Phase 1 interventional study of ALT001 in Multiple System Atrophy, sponsored by yilong Wang. Recruiting at 1 site in China. Open to participants aged 30 Years to 75 Years. Per ClinicalTrials.gov, last updated 2026-09-09.
Sponsored by yilong Wang · Early Phase 1, Interventional, and Treatment
This is an open-label, single-center, prospective, single-arm clinical study. The primary objective of this study is to evaluate the safety, tolerability, and preliminary efficacy of ALT001 in the treatment of patients with multiple system atrophy (MSA) in a real-world setting.
Multiple system atrophy (MSA) is a progressive neurodegenerative disorder characterized by a blend of autonomic dysfunction, Parkinson's syndrome, and cerebellar syndrome. The incidence of MSA ranges from 1.9 to 4.9 per 100,000 individuals, with an average age of onset of 56.2 years. Among individuals aged 50 years and older, the prevalence stands at 3.0 per 100,000. The mean age of MSA onset is 56.2 years, and the median survival ranges from 6.2 to 7.5 years. MSA often presents with severe autonomic dysfunction early in its course, impacting patient survival. Furthermore, due to difficulties in early diagnosis, rapid progression, and poor prognosis, nearly 50% of patients require a walking aid or physical assistance for ambulation within three years of motor symptom onset. Within five years, 60% of patients become wheelchair-dependent, and after six to eight years, most are completely bedridden, severely compromising their quality of life. Current symptomatic and supportive therapies fall short of meeting the treatment requirements of MSA patients. Furthermore, potential adverse effects and disease progression factors restrict the use of certain drugs, highlighting the critical need for the development of disease-modifying or neuroprotective agents to decelerate disease advancement. "ALT001, a nerve repair protein created by Darwin Origin (Hubei) Biopharmaceutical Co.LTD, is derived from cellular exosomes, a set of specific microenvironmental protein polymers secreted by stem cells under emergency conditions. It boasts selective assembly, targeted delivery, highly efficient tissue repair, exceptional safety, chemical stability, and convenient storage. Previous basic research has indicated ALT001's potential for promoting endogenous neural tissue repair, exhibiting significant neuroprotective and neurorestorative effects in animal models. Therefore, building upon the previously initiated study of ALT001 in patients with the MSA-P subtype and the forthcoming study in patients with the MSA-C subtype, we are broadening the inclusion and exclusion criteria to evaluate the safety, tolerability, and efficacy of ALT001 in the treatment of MSA in a real-world setting. This study aims to recruit 60 MSA patients aged between 30 and 75 years. All participants will receive three cycles of ALT001 treatment, with each cycle lasting 30 days. ALT001 will be administered via intravenous infusion (130 μg) during the first 14 days of each cycle. Assessments including vital signs, laboratory tests (e.g., routine blood tests, blood biochemical examinations, coagulation tests), and neurological evaluations (e.g., Unified Multiple System Atrophy Rating Scale [UMSARS] and Composite Autonomic Symptom Score [COMPASS]) will be conducted at each cycle and at 180 days post-treatment. Additionally, if patients experience new neurological symptoms or suspicious events, additional visits will be carried out, and researchers are required to submit and interpret relevant data within 72 hours of the event occurrence.The protocol of this study has been approved by the Ethics Committee of Beijing Tiantan Hospital. All participants will provide written informed consents before entering the study.
Exclusion Criteria:
Intravenous administration of ALT001 was given to each MSA patient in the intervention group, with intravenous administration on days 1 to 14, 31 to 44±3 and 61 to 74±5, and treatment was given once a day. Intravenous administration: ALT001 (130 μg/branch) was dissolved in 100 ml sodium chloride injection, which was completed in about 30-40 minutes.
Drug: ALT001
"ALT001" is a nerve repair protein developed by Darwin Start (Hubei) Biopharmaceutical Co., Ltd. It is a group of specific microenvironmental protein polymers secreted under the emergency conditions of stem cells. It has the advantages of selective assembly, targeted delivery, efficient repair of damaged tissues, high safety, chemical stability, easy storage, etc., and has a powerful neural repair function. According to the groups, patients would be treated with ALT001 via intrathecal injection or intravenous injection. Intravenous administration of ALT001 was given to each MSA patient in the intervention group, with intravenous administration on days 1 to 14, 31 to 44±3 and 61 to 74±5, and treatment was given once a day. Intravenous administration: ALT001 (130 μg/branch) was dissolved in 100 ml sodium chloride injection, which was completed in about 30-40 minutes.
The incidence of adverse events (AEs) and serious adverse events (SAEs)
Incidence of adverse events (AEs) and serious adverse events (SAEs) within 180±7 days after treatment.
Time frame: Day 180±7 after treatment
Changes in the unified multiple system atrophy rating scale (UMSARS) part scores, sum of part 1 and 2 scores
The unified multiple system atrophy rating scale (UMSARS) is composed of four subscales: UMSARS-I (12 items) rates patient-reported functional disability, UMSARS-II (14 items) assesses motor impairment based on a clinical examination, UMSARS-III records blood pressure and heart rate in the supine and standing positions, and UMSARS-IV (1 item) rates chore-based disability. Higher scores on the UMSARS indicate greater disability.
Time frame: Day 15, 45±3, 75±5, and 180±14 after treatment
Changes in the composite autonomic symptom score (COMPASS) scores
The Composite Autonomic Symptom Score (COMPASS) is a revised version of the 169-item Autonomic Symptom Profile assessing 11 domains of autonomic function, to the 31-item COMPASS, now assessing six domains: orthostatic hypotension (maximum score 10), vasomotor (maximum score 6), secretomotor (maximum score 7), gastrointestinal (maximum score 28), bladder (maximum score 9), and pupillomotor functions (maximum score 15). Scores from each component are recorded and a weighted total score is calculated. Higher scores on this scale indicate more severe clinical symptoms related to autonomic dysfunction. It is a validated, easy-to-use self-assessment instrument designed for clinical autonomic research and practice.
Time frame: Day 15, 45±3, 75±5, and 180±14 after treatment
Changes in plasma biomarkers (NFL, α-syn, GFAP)
Changes in NFL, α-syn, GFAP in plasma on days 15, 45±3, 74±5, 180±14 after treatment.
Time frame: Day 15, 45±3, 75±5, and 180±14 after treatment
Plan to share: No
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yilong Wang