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

A Multicenter, Open-label, Single-arm, Dose Escalation and Expansion, Phase I/IIa Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of SNUG01 in Adult Subjects With Amyotrophic Lateral Sclerosis (ALS)

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).

Study Overview

• Status: Not yet recruiting
• Conditions: Amyotrophic Lateral Sclerosis (ALS)
• Intervention / Treatment: Drug: SNUG01

Detailed Description

This is a 52-week, multicenter, open-label, single-arm, dose escalation and expansion Phase Ⅰ/Ⅱa study of SNUG01 in adults with ALS. Safety will be the primary focus, with secondary emphasis on immunogenicity, PK and preliminary clinical efficacy of SNUG01.

• Study Type: Interventional
• Enrollment (Estimated): 21
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Ji Shen; Phone Number: +8617274855306; Email: patient@sineugene.com

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100000
      • Peking University Third Hospital
  • Fujian
    • Fuzhou, Fujian, China, 350000
      • Fujian Medical University Union Hospital
  • Zhejiang
    • Hangzhou, Zhejiang, China, 310000
      • Second Affiliated Hospital Zhejiang University School of Medicine
• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02467
      • Massachusetts General Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

• Key Inclusion Criteria**:

  • 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.

• Key Exclusion Criteria**:

  • 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 3 Ascending Dose Levels, Single Injection by Intrathecal(IT)	Drug: SNUG01; AAV (adeno-associated virus) Gene therapy

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Immunogenicity of SNUG01	Change of anti-SG001 antibody and anti-AAV9 neutralizing antibody in serum, change of anti-AAV9 neutralizing antibody in CSF.	up to 1 years
PK of SNUG01 (Biodistribution and Viral Shedding)	SG001 protein level in CSF, viral vector DNA in serum, viral shedding in saliva, urine, and feces.	up to 1 years
Preliminary Clinical Efficacy of SNUG01	Change in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) , the Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) , the percent-predicted Forced Vital Capacity (%FVC) / percent-predicted Slow Vital Capacity (%SVC) and muscle strength, the Patient Health Questionnaire (PHQ-9) and NfL level in CSF and serum from baseline to Week 48 after administration.	up to 1 years

Collaborators and Investigators

• Sponsor: SineuGene Therapeutics Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): December 1, 2025
• Primary Completion (Estimated): March 30, 2028
• Study Completion (Estimated): September 30, 2028

Study Registration Dates

• First Submitted: August 8, 2025
• First Submitted That Met QC Criteria: September 5, 2025
• First Posted (Estimated): September 11, 2025

Study Record Updates

• Last Update Posted (Estimated): September 11, 2025
• Last Update Submitted That Met QC Criteria: September 5, 2025
• Last Verified: September 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Metabolic Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Nutritional and Metabolic Diseases; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: SNUG01-101

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No