Expansion Study of ALT001 in Patients With Multiple System Atrophy

NCT07476352 | Not Yet Recruiting Early Phase 1 DMC

• 2 other identifiers: KY2026-048-01HX-A-2025122
• Study Type: interventional
• Target: 60
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Multiple System Atrophy

Keywords

Multiple system atrophy

Outcome Measures

Primary Outcomes (2)

• 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.

  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.

  Day 15, 45±3, 75±5, and 180±14 after treatment

Secondary Outcomes (5)

• Changes in the composite autonomic symptom score (COMPASS) scores

  Day 15, 45±3, 75±5, and 180±14 after treatment

• Changes in the incidence of orthostatic hypotension

  Day 15, 45±3, 75±5, and 180±14 after treatment

• Variation of three-dimensional gait analysis parameters under multitasking

  Day 180±14 after treatment

• Changes in the EQ-5D scores

  Day 180±14 after treatment

• Changes in plasma biomarkers (NFL, α-syn, GFAP)

  Day 15, 45±3, 75±5, and 180±14 after treatment

Study Arms (1)

Intervention group

EXPERIMENTAL

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

Interventions

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.

Intervention group

Eligibility Criteria

• Age: 30 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• \. Age between 30 and 75 years inclusive, either sex;
• \. Clinically established or clinically probable MSA (including both MSA-C and MSA-P subtypes);
• \. Ability to walk independently or with the aid of a walking device for at least 10 meters;
• \. Provision of written informed consent.

You may not qualify if:

• \. Evidence of other central nervous system pathologies on brain MRI at screening suggesting a diagnosis of neurodegenerative diseases other than MSA;
• \. Presence of immune-mediated diseases that are inadequately controlled or require treatment with biologic agents;
• \. Known history of allergies to biologic agents, such as proteins or cell-based products;
• \. Receipt of any vaccination within the past 1 month;
• \. Patients with a prior definitive diagnosis of malignancy or those currently receiving anti-tumor drug therapy;
• \. Patients with a prior definitive diagnosis of epilepsy or those currently taking anti-epileptic drugs;
• \. 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;
• \. Patients with a history of alcohol or substance abuse, or alcohol or substance dependence within the past 2 years;
• \. Patients diagnosed with psychiatric disorders according to DSM-V criteria, or those with obvious suicidal intent;
• \. Patients who are pregnant, lactating, or have the potential to become pregnant, or those planning a pregnancy;
• \. Inability to comply with follow-up assessments due to other reasons;
• \. Patients deemed by the investigator to be unsuitable for participation in this study.

Sponsors & Collaborators

• yilong Wang: lead

Study Sites (1)

Beijing Tiantan Hospital

Beijing, Beijing Municipality, 100070, China

Location

MeSH Terms

Conditions

Multiple System Atrophy

Condition Hierarchy (Ancestors)

Primary Dysautonomias; Autonomic Nervous System Diseases; Nervous System Diseases; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Yilong Wang

  Beijing Tiantan Hospital, Capital Medical University, Beijing, China

  PRINCIPAL INVESTIGATOR

• Tao Feng

  Beijing Tiantan Hospital, Capital Medical University, Beijing, China

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Weiqi Chen

CONTACT

8615652813380; weiqichen@aliyun.com

Yahui Zhu

CONTACT

8613439182912; zhuyahuidr@sina.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Vice President of Beijing Tiantan Hospital

Study Record Dates

• First Submitted: March 12, 2026
• First Posted: March 17, 2026
• Study Start: May 6, 2026
• Primary Completion (Estimated): October 30, 2027
• Study Completion (Estimated): October 30, 2027
• Last Updated: March 30, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)