NCT07077668Not yet recruitingEarly phase 1
Extended Study of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation
Source: ClinicalTrials.gov · Last updated 22 Jul 2025 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Early phase 1
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 20 Jun 2025
- Start date
- 1 Jul 2025
- Primary completion date
- 31 Dec 2026
- Lead sponsor
- Beijing Tiantan Hospital
- Diseases
- ALS (motor neurone disease)
- NCT number
- NCT07077668
What you commit to
- Study type: Interventional · Early phase 1
- Planned participants: 10
- From 1 Jul 2025 to the primary completion date, 31 Dec 2026
- No sites listed in no country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patients who have received any vaccination within 28 days.
Other illness
- Patients diagnosed with other mental illnesses according to the DSM - V diagnostic criteria, or those with obvious suicidal intent.
- Patients with a history of alcohol or drug abuse.
Lab values
- 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.)
Other
- Patients with SOD1 mutation sites occurring at nucleotides 44 - 66 (counting from the start of SOD1 protein translation) and patients with P.F21C mutation.
- Pregnant, lactating patients, those with a possibility of pregnancy, or patients planning to become pregnant.
- Patients who are unable to cooperate with the follow - up for other reasons.
Who the study is looking for
- 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.
Age in the registry: 18 years to 75 years · Sex: All · Does not accept healthy volunteers
This page is information, not medical advice. Discuss any trial with your doctor.