ALS (Amyotrophic Lateral Sclerosis)Early Phase 1NCT07077668
Extended Study of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation
A Early Phase 1 study testing A sterile aqueous solution of RAG - 17 preparation (sodium salt) in people with ALS (Amyotrophic Lateral Sclerosis), run by Beijing Tiantan Hospital.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- ALS (Amyotrophic Lateral Sclerosis)
- What is being tested?
- A sterile aqueous solution of RAG - 17 preparation (sodium salt)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- No sites listed yet
- Study dates
- Jul 2025to 31 Dec 2026
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
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.
Read the 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.
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Being tested
- A sterile aqueous solution of RAG - 17 preparation (sodium salt)
- Keywords
- Amyotrophic lateral sclerosis, RAG-17, SOD1
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: A sterile aqueous solution of RAG - 17 preparation (sodium salt) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
Measured: 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
The level of SOD1 protein in the cerebrospinal fluid of ALS patients is positively correlated with the disease severity. Changes in the level of SOD1 protein in cerebrospinal fluid relative to the baseline at 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 after treating SOD1 - ALS patients with RAG - 17 in the real - world setting
Measured: 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
The level of neurofilament light chain (NFL) in blood of ALS patients. Value increase indicates neuroinflammation or nerve damage. Changes in plasma neurofilament light chain protein (NFL) levels relative to the baseline at 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 after treating SOD1 - ALS patients with RAG - 17 in the real world
Measured: 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
The time of occurrence of invasive mechanical ventilation after treating SOD1-ALS patients with RAG-17 in the real world
Measured: From date of randomization until the date of first documented event about invasive mechanical ventilation. The assessment period is up to 30 months.
Gastrostomy
The time of occurrence of gastrostomy after treating SOD1-ALS patients with RAG-17 in the real world
Measured: From date of randomization until the date of first documented event about gastrostomy. The assessment period is up to 30 months.
Death
The time of occurrence of death after treating SOD1-ALS patients with RAG-17 in the real world
Measured: From randomization date to date of death from any cause. The assessment period is up to 60 months.
Muscle strength (MRC Scale)
Medical Research Council Scale (MRC Scale) is an assessment that is used to measure the change in muscle strength. MRC Scales ranges from grade 0 to grade 5, where grade 0 means no muscle contraction, grade 5 means muscle has full strength. Changes in muscle strength relative to the baseline at 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 after treating SOD1-ALS patients with RAG-17 in the real world
Measured: 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
Change in Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) score (from 40 to 200, score decline indicates worse outcome of ALS patients, or disease progression and disability). Changes in ALSAQ-40 relative to the baseline at 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 after treating SOD1 - ALS patients with RAG - 17 in the real world
Measured: 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
The EuroQol 5 Dimension 5 Level (EQ-5D-5L) is a self-report survey that is used to measure the changes in the quality of life. EQ-5D-5L contains 5 domains including mobility, self-care, usual activities, pain/discomfort, and anxiety/depression, and each domain is scored on a 5-level severity from Level1(no difficulty) to Level5 (extreme difficulty). Changes in EQ-5D-5L relative to the baseline at 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 after treating SOD1 - ALS patients with RAG - 17 in the real world.
Measured: 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
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Jul 2025
- Study startJul 2025 (estimated)
- Primary completion31 Dec 2026 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ALS (Amyotrophic Lateral Sclerosis)Studies of the same condition, open and under way.
- More trials run by Beijing Tiantan HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Beijing Tiantan Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, yilong Wang, Beijing Tiantan Hospital
- Investigators
- Wang YiLong, Principal investigator, Beijing Tiantan Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07077668
- Sponsor's protocol ID
- HX-A-2025020
- Status last verified
- May 2025
- First submitted
- 20 Jun 2025
- Last updated
- 22 Jul 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Extended Study of RAG-17 in the Treatment of Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation
- ClinicalTrials.gov ID
- NCT07077668
- Acronym / sponsor ID
- HX-A-2025020
- Conditions
- ALS (Amyotrophic Lateral Sclerosis)
- Keywords
- Amyotrophic lateral sclerosis, RAG-17, SOD1
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Beijing Tiantan HospitalUniversity, hospital or other
- Study start
- Jul 2025 (estimated)
- Primary completion
- 31 Dec 2026 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 22 Jul 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment Group | Experimental | 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 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (7 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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. | Drug | Treatment Group |
Primary outcome
Incidence of adverse events and serious adverse events
Time frame: Baseline to 570 days
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.
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
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)
8 secondary outcomes
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Jul 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Wang YiLong principal investigator
Beijing Tiantan Hospital
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing A sterile aqueous solution of RAG - 17 preparation (sodium salt) in people with ALS (Amyotrophic Lateral Sclerosis), run by Beijing Tiantan Hospital.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 22 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Wang YiLong (13911666571, yilong528@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of adverse events and serious adverse events (time frame: Baseline to 570 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jul 2025.