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

Обзор

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.

Подробное описание

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.

Вмешательства

• Препарат A sterile aqueous solution of RAG - 17 preparation (sodium salt)
  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 RAG - 17 for 150 mg: 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.

Первичные конечные точки

• Incidence of adverse events and serious adverse events [Срок оценки: Baseline to 570 days]
• ALSFRS-R score [Срок оценки: 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, and 570±3 days]

Вторичные конечные точки (8)

• The level of SOD1 protein in cerebrospinal fluid [Срок оценки: baseline, 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, and 540±3 days]
• Plasma neurofilament light chain protein (NFL) level [Срок оценки: baseline, 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days, and 540±3 days]
• Invasive mechanical ventilation [Срок оценки: From date of randomization until the date of first documented event about invasive mechanical ventilation. The assessment period is up to 30 months.]
• Gastrostomy [Срок оценки: From date of randomization until the date of first documented event about gastrostomy. The assessment period is up to 30 months.]
• Death [Срок оценки: From randomization date to date of death from any cause. The assessment period is up to 60 months.]
• Muscle strength (MRC Scale) [Срок оценки: baseline, 60±3 days, 120±3 days, 180±3 days, 240±3 days, 300±3 days, 360±3 days, 450±3 days and 540±3]
• ALSAQ-40 [Срок оценки: 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 and 570±3 days]
• EQ-5D-5L [Срок оценки: 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 and 570±3 days]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Список центров уточняется — проверьте первичный протокол.

Идентификаторы

NCT: NCT07077668 · HX-A-2025020

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗