Clinical Trials in the USA / NCT07169175
Starting soon
Phase 1/2
A Phase Ⅰ/Ⅱa Study of SNUG01 in Adult Subjects With ALS
NCT07169175 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Amyotrophic Lateral Sclerosis (ALS)
Investigational drug(s) / intervention(s)
SNUG01: AAV (adeno-associated virus) Gene therapy
Study 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).
Eligibility
* 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 | Hangzhou | Zhejiang |
Other trials for the same condition
Study of COYA 302 for the Treatment of ALS
Active, not recruiting
Safety, PK and Biodistribution of 18F-OP-801 in Patients With ALS, AD, MS, PD and Healthy Volunteers
Recruiting
Control of Assistive Devices Via Brain-Computer Interface Technology
Enrolling by invitation
Official registry record
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.
View NCT07169175 on ClinicalTrials.gov ↗ ← All trials in the USA