Status:
NOT_YET_RECRUITING
A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS
Lead Sponsor:
SineuGene Therapeutics Co., Ltd.
Conditions:
Amyotrophic Lateral Sclerosis (ALS)
Eligibility:
All Genders
18-80 years
Phase:
PHASE1
PHASE2
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).
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 immuno...
Eligibility Criteria
Inclusion
- 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.
Exclusion
Key Trial Info
Start Date :
December 1 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 30 2028
Estimated Enrollment :
21 Patients enrolled
Trial Details
Trial ID
NCT07169175
Start Date
December 1 2025
End Date
September 30 2028
Last Update
September 11 2025
Active Locations (4)
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1
Massachusetts General Hospital
Boston, Massachusetts, United States, 02467
2
Peking University Third Hospital
Beijing, Beijing Municipality, China, 100000
3
Fujian Medical University Union Hospital
Fuzhou, Fujian, China, 350000
4
Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, China, 310000