Extended Study of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation

Capital Medical University

Status and phase: Not yet enrolling; Early Phase 1

Conditions: ALS (Amyotrophic Lateral Sclerosis)

Treatments: Drug: A sterile aqueous solution of RAG - 17 preparation (sodium salt)

Study type: Interventional

Funder types: Other

Identifiers

NCT07077668

HX-A-2025020

Details and patient eligibility

About

This study primarily evaluates the safety, tolerability, and efficacy of RAG - 17 in adult ALS patients with SOD1 - mutated genes in the real - world setting.

Full description

Based on the results of the previous investigator - initiated trial (IIT) of RAG - 17 in the treatment of SOD1 - ALS, this study further planned to conduct another small - sample real - world research. By administering regular and quantitative intrathecal injections of RAG - 17 to subjects who meet the inclusion criteria of the study, the safety, tolerability, and efficacy of RAG - 17 in adult ALS patients with SOD1 - mutated genes in the real - world setting will be evaluated.

Enrollment: 10 estimated patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• 18 ≤ Age ≤ 75 years old, regardless of gender.
• ALS patients with confirmed SOD1 gene mutations (known SOD1 mutation sites with reported relevant disease progression).
• The diagnosis meets the criteria for definite or probable familial or sporadic ALS in the El Escorial diagnostic criteria for amyotrophic lateral sclerosis revised by the World Federation of Neurology.
• The patient himself/herself or their legal representative clearly understands, voluntarily participates in this study, and signs the informed consent form.

Exclusion criteria

• Patients with SOD1 mutation sites occurring at nucleotides 44 - 66 (counting from the start of SOD1 protein translation) and patients with P.F21C mutation.
• Patients diagnosed with other mental illnesses according to the DSM - V diagnostic criteria, or those with obvious suicidal intent.
• Patients with severe hepatic insufficiency, severe renal insufficiency, or severe cardiac insufficiency. (Severe hepatic insufficiency refers to an ALT value ≥ 2.0 times the upper limit of normal or an AST value ≥ 2.0 times the upper limit of normal; severe renal insufficiency refers to a CRE ≥ 1.5 times the upper limit of normal or an eGFR < 40 mL/min/1.73m²; severe cardiac insufficiency refers to a NYHA score of 3 - 4.)
• Patients with a history of alcohol or drug abuse.
• Pregnant, lactating patients, those with a possibility of pregnancy, or patients planning to become pregnant.
• Patients who have received any vaccination within 28 days.
• Patients who are unable to cooperate with the follow - up for other reasons.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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10 participants in 1 patient group

Treatment Group

Experimental group

Description:

Patients who meet the inclusion and exclusion criteria are required to come to the hospital at the following time points to complete the intrathecal bolus injection of quantitative RAG - 17, relevant examinations, and evaluations by the research physicians: baseline, 60 ± 3 days, 120 ± 3 days, 180 ± 3 days, 240 ± 3 days, 300 ± 3 days, 360 ± 3 days, 450 ± 3 days, 540 ± 3 days. An online follow - up will be conducted at 570 ± 3 days.

Treatment:

Drug: A sterile aqueous solution of RAG - 17 preparation (sodium salt)