Gene MutationEarly Phase 1CREATIONNCT05903690

Safety and Tolerance of RAG-17 in Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation

A Early Phase 1 study testing RAG-17 in people with Gene Mutation, run by Beijing Tiantan Hospital at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 to 75 years
Conditions the study is for: Gene Mutation
What is being tested?: RAG-17
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 6 participants
Where: 1 siteChina
Study dates: 24 May 2023to 11 Jul 2024
Phase: Early Phase 1
Are there results?: None posted in the registry

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

Patients who are judged by professional medical staff to still be able to carry out the clinical trial project cycle;
18 years old ≤ age ≤ 75 years old, males or females;
ALS patients with confirmed SOD1 gene mutations document (known SOD1 mutation sites and related disease progression have been reported);
Forced vital capacity ≥ 50% of predicted vital capacity during the screening period;
Diagnosis of confirmed or probable familial or sporadic ALS in accordance with the revised EI Escorial diagnostic criteria for amyotrophic lateral sclerosis of the World Federation of Neurology;
The patient or patient's legal representative clearly understands and voluntarily participates in the study and signs the informed consent form;
Subjects (including male subjects) are willing to have no birth plan and voluntarily take effective contraceptive measures during the entire study period and within 3 months after the end of the study, and have no plan to donate sperm or eggs.

Who cannot join

Patients with SOD1 mutations occurring at nucleotides 44 to 66 (calculated from the start of SOD1 protein translation), patients with P.F21C mutation;
Patients who have previously received or are currently receiving Tofersen treatment;
HIV test positive or history of positive tests;
Positive hepatitis C virus antibody or history of positive tests;
Active hepatitis B infection (positive hepatitis B surface antigen and/or positive hepatitis B core antibody);
Have used other investigational drugs within 1 month or within 5 drug half-lives;

Show all 17Show fewer

Diseases and deformities of the lumbar spine;
Have other conditions known to be associated with motor neuron dysfunction that may confuse or obscure an ALS diagnosis;
Other psychiatric disorders diagnosed according to DSM-V diagnostic criteria, or significant suicide intent;
With severe hepatic insufficiency, renal insufficiency or severe cardiac insufficiency (severe hepatic insufficiency refers to ALT value≥2.0 times the upper limit of normal value or AST value≥2.0 times the upper limit of normal value; severe renal insufficiency refers to CRE≥1.5 times the upper limit of normal value or eGFR<40mL/min/1.73m2; severe cardiac insufficiency refers to NYHA class 3-4);
Permanently dependent on ventilator-assisted ventilation;
History of alcohol and drug abuse;
Patients who are pregnant, breast-feeding, or who are likely to become pregnant and plan to become pregnant;
Patients participating in other clinical trials or using other biological agents, drugs or devices under investigation;
Patients who have received any vaccinations within 28 days;
Contraindications to MRI (eg, claustrophobia);
Unable to be cooperative and complete the follow-up due to other reasons.

Trial locations and contacts

1 site in 1 country.

China1 site

Beijing Tiantan Hospital
Beijing, 100070

Other trials in China

About this study

The goal of this clinical trial is to evaluate the safety, tolerability and pharmacokinetics of RAG-17 in adult amyotrophic lateral sclerosis (ALS) patients with SOD1 mutation. Patients will receive drug treamtent via dose escalation which ranging from minimum of 60 mg to the maximum tolerated dose (MTD), after reaching the tolerated dose, a fixed dose of the drug is given once every two months for continuous treatment, and the total treatment cycle is 8 months. The duration of this study is two years.

Read the detailed description

This study is an open-label, single center, first-in-Human dose escalation study to evaluate the safety, tolerability and pharmacokinetics of RAG-17 in adultamyotrophic lateral sclerosis (ALS) patients with SOD1 mutation via dose escalations. Patients will receive 60mg of RAG-17 firstly, within 14 days after the first administration, the subject has no adverse event (AE) and serious adverse event (SAE), the subject can accept dose escalation every 30mg/14 days of observation period. 3 to 4 dose escalations are planned to reach the dose limiting toxicity (DLT), with optimal doses for continual 6-month treatment cycles. For the dose of subsequent continuous treatment, an optimal dose between the safe dose and the maximum tolerated dose (MTD) is selected as the continuous treatment dose. SAS software is used for safety analysis. The sample size of this study is 6 ALS patients with SOD1 mutation.

Conditions: Gene Mutation
Being tested: RAG-17
Keywords: RAG-17, SOD1 Gene Mutation, Amyotrophic Lateral Sclerosis
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.

RAG-17Drug
RAG-17 60mg is used

Study groups in NCT05903690
Study group	Role	What they receive
RAG-17	Experimental	Doses of RAG-17 will range from a minimum of 60 mg to the maximum tolerated dose (MTD). Dosing once every two weeks, starting from 60 mg, with dose escalation. After reaching the tolerated dose, a fixed dose of the drug is given once every two months for continuous treatment, and the total treatment cycle is 8 months. Receives: Drug: RAG-17

What is being measured

The primary outcome is the main question the study is designed to answer.

Adverse events (AE) and serious adverse events (SAE)
The incidence of adverse events (AE) and serious adverse events (SAE) within 240 days after the first injection of RAG-17, and number of participants with treatment-related adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE) V5.0
Measured: Within 240 days after treatment
Clinical laboratory examination index
Monitor the abnormal change of clinical laboratory Index generated from general examination results before RAG-17 treatment, and within 240 days after the first injection of RAG-17, which including blood testing and urine testing. Eventually to determine the safety and tolerability of RAG-17 on adult ALS patients.
Measured: Before RAG-17 treatment and within 240 days after treatment
Physiological Parameter-Vital Sign: Body Temperature
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including body temperature in degree Celsius.
Measured: Within 240 days after treatment
Physiological Parameter-Vital Sign: Pulse Rate
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including pulse rate in beats per minute.
Measured: Within 240 days after treatment
Physiological Parameter-Vital Sign: Respiration Rate
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including respiration rate in breaths per minute.
Measured: Within 240 days after treatment
Physiological Parameter-Vital Sign: Blood Pressure
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including blood pressure in mmHg.
Measured: Within 240 days after treatment
Physiological Parameter-Vital Signs: Height, Hip Circumference and Waist
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital signs that are measured including height, hip circumference and waist in centimeter.
Measured: Within 240 days after treatment
Physiological Parameter-Vital Signs: Weight
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including body weight in kilogram.
Measured: Within 240 days after treatment
Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) is a questionnaire-based scale that is used to measure the change in functional impairment of adult ALS patients within 240 days after RAG-17 treatment. ALSFRS-R scale measures 12 aspects of physical function, and each function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0.
Measured: Within 240 days after treatment
Neurological examinations
Incidence rate of abnormalities in neurological examinations within 240 days after RAG-17 treatment
Measured: Within 240 days after treatment
Heart circulatory system examinations
Incidence rate of abnormalities in heart circulatory system examinations within 240 days after RAG-17 treatment
Measured: Within 240 days after treatment
Respiratory system examinations
Incidence rate of abnormalities in respiratory system examinations within 240 days after RAG-17 treatment
Measured: Within 240 days after treatment
Abdominal examinations
Incidence rate of abnormalities in abdominal examinations within 240 days after RAG-17 treatment
Measured: Within 240 days after treatment
ECG results
Incidence rate of abnormalities in ECG results within 240 days after RAG-17 treatment
Measured: Within 240 days after treatment

Secondary outcomes (9)

SOD1 protein levels
Changes of SOD1 protein levels in cerebrospinal fluid from baseline on day 15, day 29, day 60, day 120, day 180 and day 240 of adult ALS patients after RAG-17 treatment
Measured: baseline, day 15, day 29, day 60, day 120, day 180 and day 240
Plasma neurofilament light (NFL) levels
Changes in plasma neurofilament light (NFL) levels from baseline on day 1, day 15, day 29, day 60, day 120, day 180 and day 240 in adult ALS patients after RAG-17 treatment
Measured: baseline, day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Pharmacokinetic Parameter changes of RAG-17 in Plasma: Maximum Observed Concentration (Cmax) on day1, day15, day29, day60, day120, day180 and day240 in adult ALS patients after RAG-17 treatment
Measured: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Pharmacokinetic Parameter changes of RAG-17 in Plasma: Time to Reach Maximum Observed Concentration (Tmax) on day1, day15, day29, day60, day120, day180 and day240 in adult ALS patients after RAG-17 treatment
Measured: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Pharmacokinetic Parameter changes of RAG-17 in Plasma: Area Under the Concentration-time Curve From Time Zero to Infinity (AUCinf) on day1, day15, day29, day60, day120, day180 and day240 in adult ALS patients after RAG-17 treatment
Measured: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Pharmacokinetic Parameter changes of RAG-17 in Plasma: Area Under the Concentration-time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) on day1, day15, day29, day60, day120, day180 and day240 in adult ALS patients after RAG-17 treatment
Measured: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Pharmacokinetic Parameter changes of RAG-17 in Plasma: Apparent Terminal Elimination Half-life (t1/2) on day1, day15, day29, day60, day120, day180 and day240 in adult ALS patients after RAG-17 treatment
Measured: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Mechanical ventilation
Time to invasive mechanical ventilation in adult ALS patients treated with RAG-17
Measured: From day1 to day240
Death
Time of death in adult ALS patients treated with RAG-17
Measured: From randomization date to date of death from any cause. The assessment period is up to 8 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

24 May 202311 Jul 2024

First posted15 Jun 2023
Study start24 May 2023
Primary completion11 Jul 2024
Study completion11 Jul 2024

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (11)Papers the sponsor has linked to this study.
Other trials for Gene MutationStudies 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: 6 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT05903690
Sponsor's protocol ID: HX-A-2023004
Acronym: CREATION
Status last verified: Apr 2025
First submitted: 7 May 2023
Last updated: 24 Apr 2025
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

Feldman EL, Goutman SA, Petri S, Mazzini L, Savelieff MG, Shaw PJ, Sobue G. Amyotrophic lateral sclerosis. Lancet. 2022 Oct 15;400(10360):1363-1380. doi: 10.1016/S0140-6736(22)01272-7. Epub 2022 Sep 15.
PubMed 36116464
Chen L, Zhang B, Chen R, Tang L, Liu R, Yang Y, Yang Y, Liu X, Ye S, Zhan S, Fan D. Natural history and clinical features of sporadic amyotrophic lateral sclerosis in China. J Neurol Neurosurg Psychiatry. 2015 Oct;86(10):1075-81. doi: 10.1136/jnnp-2015-310471. Epub 2015 Jun 29.
PubMed 26124198
Rosenbohm A, Liu M, Nagel G, Peter RS, Cui B, Li X, Kassubek J, Rothenbacher D, Lule D, Cui L, Ludolph AC; ALS Registry Swabia Study Group. Phenotypic differences of amyotrophic lateral sclerosis (ALS) in China and Germany. J Neurol. 2018 Apr;265(4):774-782. doi: 10.1007/s00415-018-8735-9. Epub 2018 Feb 1.
PubMed 29392461
Amyotrophic Lateral Sclerosis (ALS) Fact Sheet. National Institute of Neurological Disorders and Stroke. https://www.ninds.nih.gov/amyotrophic-lateral-sclerosis-als-fact-sheet#treatment. October 2022.
Miller RG, Jackson CE, Kasarskis EJ, England JD, Forshew D, Johnston W, Kalra S, Katz JS, Mitsumoto H, Rosenfeld J, Shoesmith C, Strong MJ, Woolley SC; Quality Standards Subcommittee of the American Academy of Neurology. Practice parameter update: the care of the patient with amyotrophic lateral sclerosis: drug, nutritional, and respiratory therapies (an evidence-based review): report of the Quality Standards Subcommittee of the American Academy of Neurology. Neurology. 2009 Oct 13;73(15):1218-26. doi: 10.1212/WNL.0b013e3181bc0141.
PubMed 19822872
Barrington CJ, Burruano M, Carney C, Choi J, Januska J, Lachuk L, Oskarsson B, Rodriguez C, Speicher L, Tereso G. Addressing the role of edaravone in the management of amyotrophic lateral sclerosis and gaps in care and access: expert panel recommendations. Am J Manag Care. 2021 Aug;27(12 Suppl):S231-S237. doi: 10.37765/ajmc.2021.88732.
PubMed 34382759
Emery AE, Holloway S. Familial motor neuron diseases. Adv Neurol. 1982;36:139-47. No abstract available.
PubMed 7180680
Chattopadhyay M, Valentine JS. Aggregation of copper-zinc superoxide dismutase in familial and sporadic ALS. Antioxid Redox Signal. 2009 Jul;11(7):1603-14. doi: 10.1089/ars.2009.2536.
PubMed 19271992
Shahrizaila N, Sobue G, Kuwabara S, Kim SH, Birks C, Fan DS, Bae JS, Hu CJ, Gourie-Devi M, Noto Y, Shibuya K, Goh KJ, Kaji R, Tsai CP, Cui L, Talman P, Henderson RD, Vucic S, Kiernan MC. Amyotrophic lateral sclerosis and motor neuron syndromes in Asia. J Neurol Neurosurg Psychiatry. 2016 Aug;87(8):821-30. doi: 10.1136/jnnp-2015-312751. Epub 2016 Apr 19.
PubMed 27093948
Brown RH, Al-Chalabi A. Amyotrophic Lateral Sclerosis. N Engl J Med. 2017 Jul 13;377(2):162-172. doi: 10.1056/NEJMra1603471. No abstract available.
PubMed 28700839
Chen W, Jiang L, Duan C, Kang M, Ye J, Wang L, Pan Y, Qu H, Liao X, Zhou X, Li Y, Gan Z, Chen J, Schacht I, Place RF, Li LC, Wang Y. Oligonucleotide-siRNA conjugate for SOD1 amyotrophic lateral sclerosis: a phase 1 trial. Nat Med. 2026 Jul;32(7):2619-2628. doi: 10.1038/s41591-026-04491-7. Epub 2026 Jul 15.
PubMed 42458007

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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Study at a glance

From the registry record, last updated by the sponsor on 24 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Safety and Tolerance of RAG-17 in Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation
ClinicalTrials.gov ID: NCT05903690
Acronym / sponsor ID: CREATION / HX-A-2023004
Conditions: Gene Mutation
Keywords: RAG-17, SOD1 Gene Mutation, Amyotrophic Lateral Sclerosis
Phase: Early Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 6 participants (actual)
Lead sponsor: Beijing Tiantan HospitalUniversity, hospital or other
Collaborators: Ractigen Therapeutics.
Study start: 24 May 2023 (actual)
Primary completion: 11 Jul 2024 (actual)
Study completion: 11 Jul 2024 (actual)
First posted: 15 Jun 2023 (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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
RAG-17	Experimental	Drug: RAG-17Doses of RAG-17 will range from a minimum of 60 mg to the maximum tolerated dose (MTD). Dosing once every two weeks, starting from 60 mg, with dose escalation. After reaching the tolerated dose, a fixed dose of the drug is given once every two months for continuous treatment, and the total treatment cycle is 8 months.

Who may qualify

Ages: 18 to 75 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

Patients who are judged by professional medical staff to still be able to carry out the clinical trial project cycle;
18 years old ≤ age ≤ 75 years old, males or females;
ALS patients with confirmed SOD1 gene mutations document (known SOD1 mutation sites and related disease progression have been reported);
Forced vital capacity ≥ 50% of predicted vital capacity during the screening period;
Diagnosis of confirmed or probable familial or sporadic ALS in accordance with the revised EI Escorial diagnostic criteria for amyotrophic lateral sclerosis of the World Federation of Neurology;

Key exclusion criteria (17 listed)

Patients with SOD1 mutations occurring at nucleotides 44 to 66 (calculated from the start of SOD1 protein translation), patients with P.F21C mutation;
Patients who have previously received or are currently receiving Tofersen treatment;
HIV test positive or history of positive tests;
Positive hepatitis C virus antibody or history of positive tests;
Active hepatitis B infection (positive hepatitis B surface antigen and/or positive hepatitis B core antibody);

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
RAG-17RAG-17 60mg is used	Drug	RAG-17

Primary outcome

Adverse events (AE) and serious adverse events (SAE)
Time frame: Within 240 days after treatment
The incidence of adverse events (AE) and serious adverse events (SAE) within 240 days after the first injection of RAG-17, and number of participants with treatment-related adverse events as assessed by Common Terminology Criteria for Adverse Events (CTCAE) V5.0
Clinical laboratory examination index
Time frame: Before RAG-17 treatment and within 240 days after treatment
Monitor the abnormal change of clinical laboratory Index generated from general examination results before RAG-17 treatment, and within 240 days after the first injection of RAG-17, which including blood testing and urine testing. Eventually to determine the safety and tolerability of RAG-17 on adult ALS patients.
Physiological Parameter-Vital Sign: Body Temperature
Time frame: Within 240 days after treatment
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including body temperature in degree Celsius.
Physiological Parameter-Vital Sign: Pulse Rate
Time frame: Within 240 days after treatment
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including pulse rate in beats per minute.
Physiological Parameter-Vital Sign: Respiration Rate
Time frame: Within 240 days after treatment
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including respiration rate in breaths per minute.
Physiological Parameter-Vital Sign: Blood Pressure
Time frame: Within 240 days after treatment
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including blood pressure in mmHg.
Physiological Parameter-Vital Signs: Height, Hip Circumference and Waist
Time frame: Within 240 days after treatment
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital signs that are measured including height, hip circumference and waist in centimeter.
Physiological Parameter-Vital Signs: Weight
Time frame: Within 240 days after treatment
Change in vital signs are monitored within 240 days after RAG-17 treatment to determine the safety and tolerability of RAG-17 on adult ALS patients. Vital sign that is measured including body weight in kilogram.
Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R)
Time frame: Within 240 days after treatment
Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) is a questionnaire-based scale that is used to measure the change in functional impairment of adult ALS patients within 240 days after RAG-17 treatment. ALSFRS-R scale measures 12 aspects of physical function, and each function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0.
Neurological examinations
Time frame: Within 240 days after treatment
Incidence rate of abnormalities in neurological examinations within 240 days after RAG-17 treatment
Heart circulatory system examinations
Time frame: Within 240 days after treatment
Incidence rate of abnormalities in heart circulatory system examinations within 240 days after RAG-17 treatment
Respiratory system examinations
Time frame: Within 240 days after treatment
Incidence rate of abnormalities in respiratory system examinations within 240 days after RAG-17 treatment
Abdominal examinations
Time frame: Within 240 days after treatment
Incidence rate of abnormalities in abdominal examinations within 240 days after RAG-17 treatment
ECG results
Time frame: Within 240 days after treatment
Incidence rate of abnormalities in ECG results within 240 days after RAG-17 treatment

9 secondary outcomes

SOD1 protein levels
Time frame: baseline, day 15, day 29, day 60, day 120, day 180 and day 240
Plasma neurofilament light (NFL) levels
Time frame: baseline, day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Time frame: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Time frame: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Time frame: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Time frame: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Pharmacokinetic Changes of RAG-17
Time frame: day 1, day 15, day 29, day 60, day 120, day 180 and day 240
Mechanical ventilation
Time frame: From day1 to day240
Death
Time frame: From randomization date to date of death from any cause. The assessment period is up to 8 months

Results and safety

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2025.
Background: PubMed 36116464Feldman EL, Goutman SA, Petri S, Mazzini L, Savelieff MG, Shaw PJ, Sobue G. Amyotrophic lateral sclerosis. Lancet. 2022 Oct 15;400(10360):1363-1380. doi: 10.1016/S0140-6736(22)01272-7. Epub 2022 Sep 15.
Background: PubMed 26124198Chen L, Zhang B, Chen R, Tang L, Liu R, Yang Y, Yang Y, Liu X, Ye S, Zhan S, Fan D. Natural history and clinical features of sporadic amyotrophic lateral sclerosis in China. J Neurol Neurosurg Psychiatry. 2015 Oct;86(10):1075-81. doi: 10.1136/jnnp-2015-310471. Epub 2015 Jun 29.
Background: PubMed 29392461Rosenbohm A, Liu M, Nagel G, Peter RS, Cui B, Li X, Kassubek J, Rothenbacher D, Lule D, Cui L, Ludolph AC; ALS Registry Swabia Study Group. Phenotypic differences of amyotrophic lateral sclerosis (ALS) in China and Germany. J Neurol. 2018 Apr;265(4):774-782. doi: 10.1007/s00415-018-8735-9. Epub 2018 Feb 1.
BackgroundAmyotrophic Lateral Sclerosis (ALS) Fact Sheet. National Institute of Neurological Disorders and Stroke. https://www.ninds.nih.gov/amyotrophic-lateral-sclerosis-als-fact-sheet#treatment. October 2022.
Background: PubMed 19822872Miller RG, Jackson CE, Kasarskis EJ, England JD, Forshew D, Johnston W, Kalra S, Katz JS, Mitsumoto H, Rosenfeld J, Shoesmith C, Strong MJ, Woolley SC; Quality Standards Subcommittee of the American Academy of Neurology. Practice parameter update: the care of the patient with amyotrophic lateral sclerosis: drug, nutritional, and respiratory therapies (an evidence-based review): report of the Quality Standards Subcommittee of the American Academy of Neurology. Neurology. 2009 Oct 13;73(15):1218-26. doi: 10.1212/WNL.0b013e3181bc0141.
Background: PubMed 34382759Barrington CJ, Burruano M, Carney C, Choi J, Januska J, Lachuk L, Oskarsson B, Rodriguez C, Speicher L, Tereso G. Addressing the role of edaravone in the management of amyotrophic lateral sclerosis and gaps in care and access: expert panel recommendations. Am J Manag Care. 2021 Aug;27(12 Suppl):S231-S237. doi: 10.37765/ajmc.2021.88732.
Background: PubMed 7180680Emery AE, Holloway S. Familial motor neuron diseases. Adv Neurol. 1982;36:139-47. No abstract available.
Background: PubMed 19271992Chattopadhyay M, Valentine JS. Aggregation of copper-zinc superoxide dismutase in familial and sporadic ALS. Antioxid Redox Signal. 2009 Jul;11(7):1603-14. doi: 10.1089/ars.2009.2536.
Background: PubMed 27093948Shahrizaila N, Sobue G, Kuwabara S, Kim SH, Birks C, Fan DS, Bae JS, Hu CJ, Gourie-Devi M, Noto Y, Shibuya K, Goh KJ, Kaji R, Tsai CP, Cui L, Talman P, Henderson RD, Vucic S, Kiernan MC. Amyotrophic lateral sclerosis and motor neuron syndromes in Asia. J Neurol Neurosurg Psychiatry. 2016 Aug;87(8):821-30. doi: 10.1136/jnnp-2015-312751. Epub 2016 Apr 19.
Background: PubMed 28700839Brown RH, Al-Chalabi A. Amyotrophic Lateral Sclerosis. N Engl J Med. 2017 Jul 13;377(2):162-172. doi: 10.1056/NEJMra1603471. No abstract available.
Derived from this study: PubMed 42458007Chen W, Jiang L, Duan C, Kang M, Ye J, Wang L, Pan Y, Qu H, Liao X, Zhou X, Li Y, Gan Z, Chen J, Schacht I, Place RF, Li LC, Wang Y. Oligonucleotide-siRNA conjugate for SOD1 amyotrophic lateral sclerosis: a phase 1 trial. Nat Med. 2026 Jul;32(7):2619-2628. doi: 10.1038/s41591-026-04491-7. Epub 2026 Jul 15.
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. This study is not recruiting at the moment.

YiLong Wang principal investigator

Beijing Tiantan Hospital

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing RAG-17 in people with Gene Mutation, run by Beijing Tiantan Hospital at 1 site in China.

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 7 inclusion and 17 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: finished, as of the sponsor's last update on 24 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.

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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse events (AE) and serious adverse events (SAE) (time frame: Within 240 days after treatment). The study lists 14 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05903690, which also lists 11 linked publications.

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 24 Apr 2025.