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)
SineuGene Therapeutics Co., Ltd.
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).
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.
Eligibility
- Age range
- 18–80 years
- Sex
- All
- 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…
Interventions
- DrugSNUG01
AAV (adeno-associated virus) Gene therapy
Locations (4)
- Massachusetts General HospitalBoston, Massachusetts
- Peking University Third HospitalBeijing, Beijing Municipality
- Fujian Medical University Union HospitalFuzhou, Fujian
- Second Affiliated Hospital Zhejiang University School of MedicineHangzhou, Zhejiang