Safety and Tolerance of RAG-17 in Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation

Prior treatment

• Patients who have previously received or are currently receiving Tofersen treatment;
• Have used other investigational drugs within 1 month or within 5 drug half-lives;
• Patients participating in other clinical trials or using other biological agents, drugs or devices under investigation;
• Patients who have received any vaccinations within 28 days;

Other illness

• HIV test positive or history of positive tests;
• Positive hepatitis C virus antibody or history of positive tests;
• Active hepatitis B infection (positive hepatitis B surface antigen and/or positive hepatitis B core antibody);
• Diseases and deformities of the lumbar spine;
• Have other conditions known to be associated with motor neuron dysfunction that may confuse or obscure an ALS diagnosis;
• Other psychiatric disorders diagnosed according to DSM-V diagnostic criteria, or significant suicide intent;
• History of alcohol and drug abuse;

Lab values

• With severe hepatic insufficiency, renal insufficiency or severe cardiac insufficiency (severe hepatic insufficiency refers to ALT value≥2.0 times the upper limit of normal value or AST value≥2.0 times the upper limit of normal value; severe renal insufficiency refers to CRE≥1.5 times the upper limit of normal value or eGFR\<40mL/min/1.73m2; severe cardiac insufficiency refers to NYHA class 3-4);

Other

• Patients with SOD1 mutations occurring at nucleotides 44 to 66 (calculated from the start of SOD1 protein translation), patients with P.F21C mutation;
• Permanently dependent on ventilator-assisted ventilation;
• Patients who are pregnant, breast-feeding, or who are likely to become pregnant and plan to become pregnant;
• Contraindications to MRI (eg, claustrophobia);
• Unable to be cooperative and complete the follow-up due to other reasons.

• Patients who are judged by professional medical staff to still be able to carry out the clinical trial project cycle;
• 18 years old ≤ age ≤ 75 years old, males or females;
• ALS patients with confirmed SOD1 gene mutations document (known SOD1 mutation sites and related disease progression have been reported);
• Forced vital capacity ≥ 50% of predicted vital capacity during the screening period;
• Diagnosis of confirmed or probable familial or sporadic ALS in accordance with the revised EI Escorial diagnostic criteria for amyotrophic lateral sclerosis of the World Federation of Neurology;
• The patient or patient's legal representative clearly understands and voluntarily participates in the study and signs the informed consent form;
• Subjects (including male subjects) are willing to have no birth plan and voluntarily take effective contraceptive measures during the entire study period and within 3 months after the end of the study, and have no plan to donate sperm or eggs.