A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS

NCT07169175 | Not Yet Recruiting Phase 1 FDA Drug

• 1 other identifier: SNUG01-101
• Study Type: interventional
• Target: 21
• Locations: 2 countries
• Sites: 4

Brief Summary

The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of SNUG01 in in adult subjects with Amyotrophic Lateral Sclerosis (ALS).

Conditions

Amyotrophic Lateral Sclerosis (ALS)

Outcome Measures

Primary Outcomes (2)

• The safety and tolerability of SNUG01 administered by intrathecal.

  Dose-limiting toxicity (DLT), adverse events (AEs), clinically significant changes in vital signs, physical examination, clinical laboratory tests (hematology, biochemistry, urinalysis, coagulation, cardiac enzymes, etc.) and 12-lead ECG, etc.

  up to 1 year after administration

• To recommend the optimal expansion dose of SNUG01 that demonstrates acceptable safety with maximum preliminary efficacy administered by IT.

  up to 1 years

Secondary Outcomes (3)

• Immunogenicity of SNUG01

  up to 1 years

• PK of SNUG01 (Biodistribution and Viral Shedding)

  up to 1 years

• Preliminary Clinical Efficacy of SNUG01

  up to 1 years

Study Arms (1)

3 Ascending Dose Levels, Single Injection by Intrathecal(IT)

EXPERIMENTAL

Drug: SNUG01

Interventions

SNUG01 DRUG

AAV (adeno-associated virus) Gene therapy

3 Ascending Dose Levels, Single Injection by Intrathecal(IT)

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects who are able to provide written informed consent form (ICF).
• Subjects who are males or females must be ≥ 18 years and ≤ 80 years of age at the screening visit.
• Subjects who have clinically definite ALS, clinically probable ALS, or clinically probable-laboratory supported ALS as specified in the revised version of the El Escorial World Federation of Neurology criteria.
• Subjects must have an ALS disease duration (from first symptom onset to the screening visit) ≤ 2 years.
• Subjects with a body mass index (BMI) ≥ 19 kg/m2 at the screening visit.
• Subjects whose percent-predicted Forced Vital Capacity (%FVC) is ≥ 70% or percent-predicted Slow Vital Capacity (%SVC) is ≥ 60%, adjusted for sex, age, and height at the screening visit.
• The ALSFRS-R score ≥ 30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be full marks.

You may not qualify if:

• Serum Anti-AAV9 neutralizing antibody titer ≥ 1:100.
• Current or previous exposure to gene therapy, stem cell products, and solid organ transplantation.
• Subjects who have implanted or are estimated to require a diaphragmatic pacing system during the study period.
• Any thromboembolic event, such as deep vein thrombosis, pulmonary arteriovenous embolism, and jugular vein embolism, has occurred within 6 months before the administration.
• Suffering from autoimmune diseases or ongoing immune-related therapy, except intranasal, inhalation, ocular, topical, intra-articular corticosteroid therapy or corticosteroid physiological replacement therapy.
• Active or chronic uncontrolled infection within 4 weeks before the administration, deemed unacceptable in the discretion of the investigator.
• Evidence of human immunodeficiency virus (HIV) and treponema pallidum (TP) infection, as documented by the treatment for HIV or TP, or by HIV or TP antibodies positivity at the screening visit.
• Has a positive serum pregnancy test at screening (females of childbearing potential only), a positive urine or serum pregnancy test at baseline (Day -1. females of childbearing potential only), or is nursing.

Sponsors & Collaborators

• SineuGene Therapeutics Co., Ltd.: lead

Study Sites (4)

Massachusetts General Hospital

Boston, Massachusetts, 02467, United States

Location

Peking University Third Hospital

Beijing, Beijing Municipality, 100000, China

Location

Fujian Medical University Union Hospital

Fuzhou, Fujian, 350000, China

Location

Second Affiliated Hospital Zhejiang University School of Medicine

Hangzhou, Zhejiang, 310000, China

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Central Study Contacts

Ji Shen

CONTACT

+8617274855306; patient@sineugene.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 8, 2025
• First Posted: September 11, 2025
• Study Start: December 1, 2025
• Primary Completion (Estimated): March 30, 2028
• Study Completion (Estimated): September 30, 2028
• Last Updated: September 11, 2025

Record last verified: 2025-09

Locations

US (1); CN (3)