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).
Eligibility
Sex
ALL
Min age
18 Years
Max age
80 Years
Healthy volunteers
No
* 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.
Primary outcome measure(s)
The safety and tolerability of SNUG01 administered by intrathecal. — up to 1 year after administration 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.
To recommend the optimal expansion dose of SNUG01 that demonstrates acceptable safety with maximum preliminary efficacy administered by IT. — up to 1 years
Trial sites (4)
Facility
City
Region
Status
Massachusetts General Hospital
Boston
Massachusetts
Peking University Third Hospital
Beijing
Beijing Municipality
Fujian Medical University Union Hospital
Fuzhou
Fujian
Second Affiliated Hospital Zhejiang University School of Medicine
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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