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

NCT07077668 | Not Yet Recruiting Early Phase 1 DMC

• 1 other identifier: HX-A-2025020
• Study Type: interventional
• Target: 10
• Locations: 0 countries
• Sites: N/A

Brief Summary

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.

Conditions

ALS (Amyotrophic Lateral Sclerosis)

Keywords

Amyotrophic lateral sclerosis; RAG-17; SOD1

Outcome Measures

Primary Outcomes (2)

• Incidence of adverse events and serious adverse events

  Incidence of adverse events and serious adverse events within 570 days after treating SOD1 - ALS patients with RAG - 17 in the real world, and number of participants with treatment-related adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE) V5.0.

  Baseline to 570 days

• ALSFRS-R score

  Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score (ALSFRS-R scores from 0 to 48, score decline indicates worse outcome of ALS patients, or disease progression and disability)

  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 Outcomes (8)

• The level of SOD1 protein in cerebrospinal fluid

  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

  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

  From date of randomization until the date of first documented event about invasive mechanical ventilation. The assessment period is up to 30 months.

• Gastrostomy

  From date of randomization until the date of first documented event about gastrostomy. The assessment period is up to 30 months.

• Death

  From randomization date to date of death from any cause. The assessment period is up to 60 months.

Study Arms (1)

Treatment Group

EXPERIMENTAL

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.

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

Interventions

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

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.

Treatment Group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• ≤ 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Beijing Tiantan Hospital: lead

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Wang YiLong

  Beijing Tiantan Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Wang YiLong

CONTACT

13911666571; yilong528@gmail.com

Chen WeiQI

CONTACT

15652813380; weiqichen@aliyun.com

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice-President of Beijing Tiantan Hospital,Chief Scientist of Neurology Center

Study Record Dates

• First Submitted: June 20, 2025
• First Posted: July 22, 2025
• Study Start: July 1, 2025
• Primary Completion (Estimated): December 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: July 22, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share