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Expansion Study of ALT001 in Patients With Multiple System Atrophy

ConditionMultiple System Atrophy
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age30-75
Sponsoryilong Wang

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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;

Trial design

Design model

Single group

Treatments tested in this trial

  • ALT001

    Drug

    "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.

Treatment groups

60 Participants
are divided into 1 treatment group
Group A: Intervention groupExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

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
2

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

Secondary outcomes

1

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
2

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

yilong Wang

Lead sponsor

Beijing Tiantan Hospital

Sponsor institution

Source ClinicalTrials.gov
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