The Study of Safety and Preliminary Efficacy of ALT001 in Patients With MultIple System Atrophy-Cerebellar Type

NCT07514923 | Recruiting Early Phase 1 DMC

• 2 other identifiers: KY2026-049-01HX-A-2025121
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 2

Brief Summary

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).

Conditions

Multiple System Atrophy - Cerebellar Subtype (MSA-C)

Keywords

Multiple system atrophy

Outcome Measures

Primary Outcomes (2)

• The incidence of investigator-reported adverse events (AEs) and serious adverse events (SAEs)

  The incidence of investigator-reported adverse events (AEs) and serious adverse events (SAEs)

  Day 90±7 after randomization

• The incidence of changes in clinical laboratory test parameters, changes in vital signs, neurological examination abnormalities and ECG abnormalities

  Laboratory tests include: routine blood test, coagulation test, blood biochemical examination, urinalysis (note that abnormal changes in laboratory test results may need to be reviewed to further determine and assess their relevance to the study). Vital signs include: abnormal body temperature (≥38℃ or ≤35℃), blood pressure (systolic blood pressure ≥180mmHg or \<90mmHg), respiration (\>24 beats/min and other rhythm abnormalities), and heart rate (\>100 beats/min or \<60 beats/min).

  Day 90±7 after randomization

Secondary Outcomes (7)

• 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, 90±7, 180±14, and 360±14 after randomization

• Changes in the scale for the assessment and rating of ataxia (SARA)

  Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization

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

  Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization

• Changes in the incidence of orthostatic hypotension

  Day 15, 45±3, 75±5, 90±7, 180±14, and 360±14 after randomization

• Changes in quick MSA quality of life questionnaire (MSA-QoL)

  Day 90±7, 180±14, and 360±14 after randomization

Other Outcomes (6)

• Changes in postvoid residual urine volume (bladder ultrasound)

  Day 90±7, 180±14, and 360±14 after randomization

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

  Day 15, 30±3, 60±5, 90±7, 180±14, and 360±14 after randomization

• Changes in cerebrospinal fluid biomarkers (α-Syn SAA, NFL, α-syn, GFAP)

  Day 31±3 and 61±5 after randomization

Study Arms (2)

Intervention group

EXPERIMENTAL

Intrathecal administration combined with intravenous administration of ALT001 was given to each MSA patient in the intervention group, with intrathecal administration on days 1, 31 and 61 and intravenous administration on days 2 to 14, 32 to 44±3 and 62 to 74±5, and treatment was given once a day. intrathecal administration: 8 mL sodium chloride injection + ALT001 (130 μg/branch) for intrathecal administration, which was completed in about 5 minutes; intravenous administration: ALT001 (130 μg/branch) was dissolved in 100 ml sodium chloride injection, which was completed in about 30-40 minutes. After completion of the above treatments and 90±7 days of follow-up, all subjects entered the OLE study phase. During this period, all participants were given the option of continuing to receive 3 phases (on Days 91 to 104±7, Days 121 to 134±7, and Days 151 to 164±7) of intravenous ALT001 administration (14 consecutive days of ALT001 130 μg qd i.v. in each phase).

Drug: ALT001

No intervention group

NO INTERVENTION

Each MSA-C patient in the blank control group received only basic treatment with no trial-related treatment. After completion of the above treatments and 90±7 days of follow-up, all subjects entered the OLE study phase. During this period, all participants were given the option of continuing to receive 3 phases (on Days 91 to 104±7, Days 121 to 134±7, and Days 151 to 164±7) of intravenous ALT001 administration (14 consecutive days of ALT001 130 μg qd i.v. in each phase).

Interventions

ALT001 DRUG

Intrathecal administration combined with intravenous administration of ALT001 was given to each MSA patient in the intervention group, with intrathecal administration on days 1, 31 and 61 and intravenous administration on days 2 to 14, 32 to 44±3 and 62 to 74±5, and treatment was given once a day. intrathecal administration: 8 mL sodium chloride injection + ALT001 (130 μg/branch) for intrathecal administration, which was completed in about 5 minutes; intravenous administration: ALT001 (130 μg/branch) was dissolved in 100 ml sodium chloride injection, which was completed in about 30-40 minutes. After completion of the above treatments and 90±7 days of follow-up, all subjects entered the OLE study phase. During this period, all participants were given the option of continuing to receive 3 phases (on Days 91 to 104±7, Days 121 to 134±7, and Days 151 to 164±7) of intravenous ALT001 administration (14 consecutive days of ALT001 130 μg qd i.v. in each phase).

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-C as defined by the 2022 MDS diagnostic criteria for multiple system atrophy;
• \. Duration of MSA-C-related motor symptoms, including ataxia, no longer than 5 years;
• \. Score ≤ 3 on Item 14 of the Unified Multiple System Atrophy Rating Scale (UMSARS) Part II (motor examination);
• \. Estimated life expectancy ≥ 1 year as assessed by the investigator;
• \. Voluntary participation in this study and provision of written informed consent

You may not qualify if:

• \. 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);
• \. 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;
• \. 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;
• \. Presence of lumbar spine diseases or deformities, or other contraindications to lumbar puncture;
• \. Coagulation abnormalities at enrollment (e.g., platelet count \<100 × 10⁹/L; prothrombin time \[PT\] \>3 seconds), a prior diagnosis of coagulation disorders such as hemophilia, or current use of two or more antiplatelet agents;
• \. Contraindications to MRI (e.g., claustrophobia, implanted cardiac pacemaker, or ferromagnetic metal);
• \. 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;
• \. Active hepatitis B infection (positive for hepatitis B surface antigen, recurrent/persistent ALT elevation, positive serum HBV DNA, or serum HBV DNA \>2 × 10⁵ IU/mL);
• \. Positive for hepatitis C virus antibody or a history of positive test results;
• \. Positive for HIV or a history of positive test results;
• \. Patients with a history of alcohol or substance abuse, or alcohol or substance dependence within the past 2 years;

Sponsors & Collaborators

• yilong Wang: lead

Study Sites (2)

Beijing Tiantan Hospital

Beijing, Beijing Municipality, 100070, China

RECRUITING

Beijing Tiantan Hospital

Beijing, Beijing Municipality, 100070, China

RECRUITING

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

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• 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: April 7, 2026
• Study Start: June 10, 2026
• Primary Completion (Estimated): January 31, 2027
• Study Completion (Estimated): October 30, 2027
• Last Updated: September 9, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (2)