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Not yet recruiting NCT07077668

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

Early Phase I Interventional ALS (Amyotrophic Lateral Sclerosis)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: A sterile aqueous solution of RAG - 17 preparation (sodium salt).
Who it may be relevant to
Registry conditions: ALS (Amyotrophic Lateral Sclerosis). Basic parameters: 18 years — 75 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Overview

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.

Detailed 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.

Interventions

  • Drug 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.

Primary outcome measures

  • Incidence of adverse events and serious adverse events [Time frame: Baseline to 570 days]
  • ALSFRS-R score [Time frame: 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]
Secondary outcome measures (8)
  • The level of SOD1 protein in cerebrospinal fluid [Time frame: 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 [Time frame: 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 [Time frame: From date of randomization until the date of first documented event about invasive mechanical ventilation. The assessment period is up to 30 months.]
  • Gastrostomy [Time frame: From date of randomization until the date of first documented event about gastrostomy. The assessment period is up to 30 months.]
  • Death [Time frame: From randomization date to date of death from any cause. The assessment period is up to 60 months.]
  • Muscle strength (MRC Scale) [Time frame: 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 [Time frame: 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 [Time frame: 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]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07077668 · HX-A-2025020

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗