Amyotrophic Lateral SclerosisEarly Phase 1NCT06454682
An IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With ALS
A Early Phase 1 study testing RJK002 Intrathecal injection in people with Amyotrophic Lateral Sclerosis, run by RJK Biopharma Ltd at 1 site in China.
Trial summary
The goal of this clinical trial is to evaluate the safety and efficacy of a single intrathecal injection of RJK002 in patients with Amyotrophic Lateral Sclerosis (ALS). The main questions it aims to answer are: The safety, tolerability, and preliminary efficacy of a single intrathecal injection of RJK002 in subjects… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- RJK002 Intrathecal injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- 11 Sep 2023to 2 Aug 2031
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Female or male subjects who are ≥ 18 years of age at screening;
- Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN);
- The duration of the disease from the first symptom (any ALS symptom) prior to the screening visit must be more than 6 months and less than 2 years (inclusive);
- The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score ≥30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be full marks;
- The forced vital capacity (FVC) of predicted during the screening period is ≥70% at screening;
- Body mass index (BMI) greater than 18 kg/m2 at screening;
- Subjects who had not taken riluzole, edaravone, or who had been on a stable dose for ≥30 days at screening and continue to take it during the study period (those who have stopped taking riluzole, edaravone for ≥ 30 days at screening may also be included, in the opinion of the Investigator);
- Understand and comply with the trial procedure, voluntarily participate in and be willing to undergo genetic testing, and sign the informed consent (the informed consent is voluntarily signed by the subject or the subject's leagally authorized representative in the case that a subject is legally incapable of providing informed consent).
Who cannot join
- Subjects with other neurological diseases similar to ALS that affect the evaluation of drug efficacy, such as cervical spondylotic myelopathy, syringomyelia, spinal cord and brain stem tumors, hirayama disease, multifocal motor neuropathy, multiple sclerosis, Guillain-Barre syndrome, Parkinson's disease and dementia;
- Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;
- Subjects with a history of spinal surgery within the last six months after the onset of ALS;
- By cytological analysis at screening, the titer of anti-AAV9 neutralizing antibody was positive (>1:100);
- AST, ALT, bilirubin, glutamyltransferase, or glutamate dehydrogenase >1.5 × ULN or serum creatinine (Scr) > ULN at screening;
- Any medical instability deemed by the Investigator to be clinically significant, including but not limited to the presence of cardiovascular and cerebrovascular, hepatic or renal dysfunction, endocrine, psychiatric, or nervous system abnormalities that the investigator believes would interfere with the overall safety or efficacy of the study;
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- Subjects with history of convulsive seizures or epilepsy (excluding febrile convulsive seizures in childhood);
- History of autoimmune diseases (excluding thyroid diseases), myelodysplastic or myeloproliferative diseases, leukemia or lymphoma, or severe scoliosis;
- Subjects who are hospitalized for surgery, pulmonary events, or nutritional support within 2 months prior to the screening period or who planned to undergo hospitalization for major surgery during the study period;
- Presence of contraindications to lumbar puncture (including, but not limited to, signs or symptoms of skin infection at the administration site and elevated intracranial pressure), receipt of any active intrathecal therapy, presence of implantable shunt tubes for draining cerebrospinal fluid (CSF), presence of implantable central nervous system (CNS) cannula, or any condition that interferes with CSF collection;
- Screening for a record of attempted suicide in the six months prior to the screening visit, who indicated four or five categories of suicidal thoughts on the Columbia-Suicide Severity Rating Suicide Scale (C-SSRS) assessment, or who the Investigator judged to be at risk of suicide attempts;
- Active infection requiring systemic antiviral or antibacterial therapy at any time between 4 weeks prior to the start of screening and dosing; Active but untreated viral or bacterial infection at any time between 4 weeks prior to the start of screening and dosing; Any febrile illness developed during the 2 weeks prior to the start of screening until treatment was given;
- Suspect or have a history of alcohol or drug abuse;
- Hepatitis B virus surface antigen, hepatitis C virus antibody, syphilis, or human immunodeficiency virus antibody positive at screening;
- Pregnant or lactating female subjects, or subjects of childbearing potential (including heterosexual male subjects and their fertile female partners), who have pregnancy plans during the study period or do not want to use effective contraception during the study period;
- Subjects who have been received any stem cell therapy or gene therapy prior to screening;
- Subjects vaccinated within 2 weeks prior to screening or expected to be vaccinated during the study period;
- Subjects with concomitant or ongoing immunosuppressive therapy within 90 days prior to IP administration;
- Subjects who have participated in another clinical trial and received any investigational therapy within 3 months prior to screening;
- Subjects who are deemed inappropriate by the investigator to participate in this clinical trial.
Trial locations and contacts
1 site in 1 country.
China1 site
The First Affiliated Hospital Fujian Medical University
Fuzhou, Fujian
About this study
Official title: A Single-site, Single-arm, Open-label, IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of AAV-RJK002 in Patients With Amyotrophic Lateral Sclerosis (ALS)
The goal of this clinical trial is to evaluate the safety and efficacy of a single intrathecal injection of RJK002 in patients with Amyotrophic Lateral Sclerosis (ALS). The main questions it aims to answer are:
- The safety, tolerability, and preliminary efficacy of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS)
- The adeno-associated virus (AAV) viral load, changes of biomarkers in serum and cerebrospinal fluid (CSF), and electromyography (EMG) motor unit counts in subjects with ALS treated with a single intrathecal injection of RJK002.
Participants will receive a single intrathecal administration of investigational product and a systemic immunomodulatory regimen. There will be 3 cohorts: 3E13 vg/person (3 mL), 6E13vg/person (6 mL), and 1.2E14 vg/person (12 mL). 3 subjects will be enrolled in each dose cohort. The dose level will be escalated sequentially from low to high.
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- RJK002 Intrathecal injection
- 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.
RJK002 Intrathecal injectionDrug
Eligible subjects will receive a single intrathecal administration of investigational product with dose 3E13 vg/person, 6E13 vg/person, and 1.2E14 vg/person sequentially.
| Study group | Role | What they receive |
|---|---|---|
| RJK002 Intrathecal injection | Experimental | Not described Receives: Drug: RJK002 Intrathecal injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
incidence of adverse events (AEs)/serious adverse events (SAEs)
Measured: Each visit within 5 years after administration
Dose limiting toxicities (DLT)
Measured as any drug-related serious adverse event that meet DLT standard. If a dose has less than 33% DLTs it will be considered tolerable.
Measured: 28 days after administration
Secondary outcomes (1)
Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score.
The ALSFRSr, a questionnaire-based scale assessing daily living function ranging from 48 (best score) to 0 (worst), was administered to the patient, or to a proxy if the patient could not communicate effectively. Decline was defined as ALSFRSr at baseline minus ALSFRSr at month 9. A positive value indicates worsening.
Measured: 28 days, 56 days, 84 days, 6 months, 12months, 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jun 2024
- Study start11 Sep 2023
- Primary completion21 Jun 2025
- Study completion2 Aug 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- 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
- Model description
- Eligible subjects will receive a single intrathecal administration of investigational product. 9 subjects will be involved in 3 doses cohort from low to high: 3E13 vg/person, 6E13 vg/person, and 1.2E14 vg/person, 3 subjects for each cohort.
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- RJK Biopharma Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06454682
- Sponsor's protocol ID
- RJK-002-I
- Status last verified
- Aug 2025
- First submitted
- 3 Jun 2024
- Last updated
- 28 Aug 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Some of data are planned to be shared on Nov 2025 and Nov 2026.
Study at a glance
From the registry record, last updated by the sponsor on 28 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-site, Single-arm, Open-label, IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of AAV-RJK002 in Patients With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT06454682
- Acronym / sponsor ID
- RJK-002-I
- Conditions
- Amyotrophic Lateral Sclerosis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- RJK Biopharma LtdIndustry (pharma, biotech, devices)
- Study start
- 11 Sep 2023 (actual)
- Primary completion
- 21 Jun 2025 (actual)
- Study completion
- 2 Aug 2031 (estimated)
- First posted
- 12 Jun 2024 (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.
The goal of this clinical trial is to evaluate the safety and efficacy of a single intrathecal injection of RJK002 in patients with Amyotrophic Lateral Sclerosis (ALS). The main questions it aims to answer are:
- The safety, tolerability, and preliminary efficacy of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS)
- The adeno-associated virus (AAV) viral load, changes of biomarkers in serum and cerebrospinal fluid (CSF), and electromyography (EMG) motor unit counts in subjects with ALS treated with a single intrathecal injection of RJK002.
Participants will receive a single intrathecal administration of investigational product and a systemic immunomodulatory regimen. There will be 3 cohorts: 3E13 vg/person (3 mL), 6E13vg/person (6 mL), and 1.2E14 vg/person (12 mL). 3 subjects will be enrolled in each dose cohort. The dose level will be escalated sequentially from low to high.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Eligible subjects will receive a single intrathecal administration of investigational product. 9 subjects will be involved in 3 doses cohort from low to high: 3E13 vg/person, 6E13 vg/person, and 1.2E14 vg/person, 3 subjects for each cohort.
| Arm | Type | Receives |
|---|---|---|
| RJK002 Intrathecal injection | Experimental | Drug: RJK002 Intrathecal injection |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Female or male subjects who are ≥ 18 years of age at screening;
- Patients with a diagnosis consistent with clinically or laboratory-supported possible, probable, or definite sporadic or familial ALSALS in accordance with Revised EI Escorial diagnostic criteria published by the World Federation of Neurology (WFN);
- The duration of the disease from the first symptom (any ALS symptom) prior to the screening visit must be more than 6 months and less than 2 years (inclusive);
- The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) score ≥30 during the screening period, and the three respiratory scores (dyspnea, upright respiration, and respiratory insufficiency) must be full marks;
- The forced vital capacity (FVC) of predicted during the screening period is ≥70% at screening;
Key exclusion criteria (20 listed)
- Subjects with other neurological diseases similar to ALS that affect the evaluation of drug efficacy, such as cervical spondylotic myelopathy, syringomyelia, spinal cord and brain stem tumors, hirayama disease, multifocal motor neuropathy, multiple sclerosis, Guillain-Barre syndrome, Parkinson's disease and dementia;
- Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;
- Subjects with a history of spinal surgery within the last six months after the onset of ALS;
- By cytological analysis at screening, the titer of anti-AAV9 neutralizing antibody was positive (>1:100);
- AST, ALT, bilirubin, glutamyltransferase, or glutamate dehydrogenase >1.5 × ULN or serum creatinine (Scr) > ULN at screening;
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 |
|---|---|---|
| RJK002 Intrathecal injectionEligible subjects will receive a single intrathecal administration of investigational product with dose 3E13 vg/person, 6E13 vg/person, and 1.2E14 vg/person sequentially. | Drug | RJK002 Intrathecal injection |
Primary outcome
incidence of adverse events (AEs)/serious adverse events (SAEs)
Time frame: Each visit within 5 years after administration
Dose limiting toxicities (DLT)
Time frame: 28 days after administration
Measured as any drug-related serious adverse event that meet DLT standard. If a dose has less than 33% DLTs it will be considered tolerable.
1 secondary outcome
Change in the ALS Functional Rating Scale-revised (ALSFRSr) Score.
Time frame: 28 days, 56 days, 84 days, 6 months, 12months, 18 months
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 28 Aug 2025.
- Individual participant dataSharing planned: Yes. Some of data are planned to be shared on Nov 2025 and Nov 2026.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 RJK002 Intrathecal injection in people with Amyotrophic Lateral Sclerosis, run by RJK Biopharma Ltd 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 8 inclusion and 20 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: under way, not looking for participants, as of the sponsor's last update on 28 Aug 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?
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?
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?
incidence of adverse events (AEs)/serious adverse events (SAEs) (time frame: Each visit within 5 years after administration). 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 28 Aug 2025.