Amyotrophic lateral sclerosis
Conditions
Interventions
Experimental group:SNUG01
Sponsors
The Second Affiliated Hospital of Zhejiang University School of Medicine
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: (1) Be able to understand and voluntarily provide written informed consent, which must be signed before performing any trial-specified study procedures; (2) Age of 18-80 years old (including the cut-off value), regardless of gender; (3) Diagnosis in accordance with the revised El Escorial criteria for the diagnosis of amyotrophic lateral sclerosis (ALS) clinically probable and supported by auxiliary examination or clinically probable or clinically confirmed ALS (see Appendix 1); (4)Patients had to have a duration of less than or equal to 36 months from the first symptom onset (any ALS symptom) to the screening visit; (5)Body mass index (BMI) >=16 kg/m2; (6)The total score of the Amyotrophic Sclerosis functional Rating scale-revised (ALSFRS-R) ranged from 15 to 35 (including the cutoff value) at screening (see Appendix 2); (7) Adequate organ function: 1) Hematology: neutrophil count >=1.0×109/L, platelet count >=80×109/L, hemoglobin >=90g/L; 2) Renal function: serum creatinine <= 2× upper limit of normal (ULN); 3) Liver function: total bilirubin (TBIL), alanine aminotransferase (ALT), aspartate aminotransferase (AST) <=2×ULN, alkaline phosphatase (ALP) <=3×ULN; 4) Coagulation function: INR, prothrombin time (PT), activated partial thromboplastin time (APTT) <= 2×ULN; 8) Participants were not currently receiving riluzole or had been receiving a stable dose of riluzole for at least 4 weeks before the screening visit. Subjects who received riluzole were expected to maintain the same dose throughout the study; (9) Subjects were not currently receiving edaravone or were receiving an approved standard edaravone regimen. Subjects who were receiving edaravone had to have completed at least one 28-day course of standard therapy before the screening visit and were expected to continue receiving edaravone throughout the study. Women of reproductive age must have a negative blood pregnancy test at screening and be nonlactating. Women of reproductive age (premenopausal women and women within 2 years of menopause, except those who had undergone bilateral tubal ligation, complete oophorectomy, or hysterectomy). Male participants whose partner was a woman of reproductive age had to agree to use an effective method of contraception (e.g., abstinence, double-barrier methods of contraception, condoms, and nonpharmacologic contraceptive methods such as intrauterine devices) for the entire duration of the study and not to donate sperm or eggs.
Exclusion criteria
Exclusion criteria: (1) At the time of screening, the serum anti - AAV9 neutralizing antibody (NAb) titer is =1:1000. (2) During the screening period, there are contraindications for lumbar puncture (including but not limited to skin infection at the administration site and signs or symptoms of increased intracranial pressure), receiving any active intrathecal treatment, having an implanted shunt for cerebrospinal fluid (CSF) drainage, having an implanted central nervous system (CNS) cannula, or having any condition that hinders CSF collection. (3) There are other diseases related to motor neuron dysfunction (progressive bulbar palsy, primary lateral sclerosis, cervical spondylosis, lumbar spondylosis, idiopathic inflammatory myopathy, etc.) that may confound or mask the diagnosis of ALS. (4) History of invasive ventilation or tracheotomy due to ALS disease, or current use of non - invasive ventilation support for an average of >=16 hours per day. (5) Presence of any febrile illness or vaccination within 7 days before the first dose. (6) Suffering from active or uncontrolled infection (including but not limited to infectious pneumonia, sepsis, herpes zoster infection) within 4 weeks before the first dose, or having a history of chronic bacterial infection that is considered unacceptable according to the judgment of the investigator. (7) Having undergone major surgery within 4 weeks before the first dose. (8) Known allergy to the drugs to be used in the treatment or their excipients. (9) Positive result in hepatitis B surface antigen (HBsAg) test (however, if the peripheral blood hepatitis B virus deoxyribonucleic acid (HBV DNA) is below the lower limit of the reference value range, the subject is eligible for inclusion); positive results in both hepatitis C virus (HCV) antibody and HCV RNA tests, positive syphilis spirochete antibody, positive human immunodeficiency virus (HIV) antibody. (10)At the time of screening, there are other serious physical or mental diseases or abnormal laboratory test results that may increase the risk of participating in the study, or interfere with the study results, as well as patients who are considered by the investigator to be unfit to participate in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety and tolerability ; | — |
Secondary
| Measure | Time frame |
|---|---|
| Immunogenicity;Efficacy; | — |
Countries
China
Contacts
Public ContactZhiying Wu
The Second Affiliated Hospital of Zhejiang University School of Medicine
Outcome results
None listed