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.
Study summary
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.
Eligibility
Sex
ALL
Min age
30 Years
Max age
75 Years
Healthy volunteers
No
Inclusion Criteria:
* 1\. Age between 30 and 75 years inclusive, either sex;
* 2\. Clinically established or clinically probable MSA (including both MSA-C and MSA-P subtypes);
* 3\. Ability to walk independently or with the aid of a walking device for at least 10 meters;
* 4\. Provision of written informed consent.
Exclusion Criteria:
* 1\. Evidence of other central nervous system pathologies on brain MRI at screening suggesting a diagnosis of neurodegenerative diseases other than MSA;
* 2\. 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);
* 3\. Presence of immune-mediated diseases that are inadequately controlled or require treatment with biologic agents;
* 4\. Known history of allergies to biologic agents, such as proteins or cell-based products;
* 5\. Receipt of any vaccination within the past 1 month;
* 6\. Patients with a prior definitive diagnosis of malignancy or those currently receiving anti-tumor drug therapy;
* 7\. Patients with a prior definitive diagnosis of epilepsy or those currently taking anti-epileptic drugs;
* 8\. Concurrent severe hepatic insufficiency, renal insufficiency, or severe cardiac insufficiency (severe hepatic insufficiency defined as ALT ≥1.5 times the upper limit of normal or AST ≥1.5 times the upper limit of normal; severe renal insufficiency defined as serum creatinine \[CRE\] ≥1.5 times the upper limit of normal or estimated glomerular filtration rate \[eGFR\] \<40 mL/min/1.73 m²; severe cardiac insufficiency defined as NYHA class 3-4), or any significant abnormalities on physical examination, vital signs, laboratory tests, or electrocardiogram that, in the investigator's opinion, require further examination or treatment, or may interfere with the study procedures or safety;
* 9\. Patients with a history of alcohol or substance abuse, or alcohol or substance dependence within the past 2 years;
* 10\. Patients diagnosed with psychiatric disorders according to DSM-V criteria, or those with obvious suicidal intent;
* 11\. Patients who are pregnant, lactating, or have the potential to become pregnant, or those planning a pregnancy;
* 12\. Inability to comply with follow-up assessments due to other reasons;
* 13\. Patients deemed by the investigator to be unsuitable for participation in this study.
Primary outcome measure(s)
The incidence of adverse events (AEs) and serious adverse events (SAEs) — Day 180±7 after treatment Incidence of adverse events (AEs) and serious adverse events (SAEs) within 180±7 days after treatment.
Changes in the unified multiple system atrophy rating scale (UMSARS) part scores, sum of part 1 and 2 scores — Day 15, 45±3, 75±5, and 180±14 after treatment 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.
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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