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).
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
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
21
AAV (adeno-associated virus) Gene therapy
Massachusetts General Hospital
Boston, Massachusetts, United States
Peking University Third Hospital
Beijing, Beijing Municipality, China
Fujian Medical University Union Hospital
Fuzhou, Fujian, China
Second Affiliated Hospital Zhejiang University School of Medicine
Hangzhou, Zhejiang, China
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.
Time frame: 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.
Time frame: up to 1 years
Immunogenicity of SNUG01
Change of anti-SG001 antibody and anti-AAV9 neutralizing antibody in serum, change of anti-AAV9 neutralizing antibody in CSF.
Time frame: 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.
Time frame: 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.
Time frame: up to 1 years
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