Amyotrophic Lateral Sclerosis AlsPhase 1NCT06493279
Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis
A Phase 1 study testing RJK002 Intrathecal injection in people with Amyotrophic Lateral Sclerosis Als, run by RJK Biopharma Ltd at 1 site in China.
Trial summary
The goal of the study aims to evaluate the safety and tolerability of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS), and to determine the recommended Phase II dose (RP2D). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis Als
- 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
- 24 Sep 2024to 30 Nov 2030
- Phase
- 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
Main Inclusion Criteria:
- 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 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 ≥3;
- 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;
Main Exclusion Criteria:
- 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;
- 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);
- Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Single-arm, Open-label, Phase I Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis (ALS)
The goal of the study aims to evaluate the safety and tolerability of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS), and to determine the recommended Phase II dose (RP2D).
- Conditions
- Amyotrophic Lateral Sclerosis Als
- 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 3E13vg (3ml), 6E13vg(6ml), and 1.2E14 vg(12ml) per person sequentially.
| Study group | Role | What they receive |
|---|---|---|
| RJK002 Intrathecal injection | Experimental | Eligible subjects will receive a single intrathecal administration of investigational product. 9 subjects will be involved in 3 doses cohort from low to high: 6E13 vg/person, 1.2E14 vg/person, and 2.4E14 vg/person, 3 subjects for each cohort. 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 after administration
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2024
- Study start24 Sep 2024
- Primary completion30 Nov 2026 (estimated)
- Study completion30 Nov 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral Sclerosis AlsStudies 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
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (estimated)
Sponsor and investigators
- Lead sponsor
- RJK Biopharma Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Peking University Third Hospital
- Responsible party
- Sponsor
- Investigators
- Dongsheng Fan, PhD, Principal investigator, Peking University Third Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06493279
- Sponsor's protocol ID
- RJK002-0001-R
- Status last verified
- Nov 2025
- First submitted
- 2 Jul 2024
- Last updated
- 20 Nov 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Part of the data are planed to share on Oct 2025, Oct 2026, and Sep 2032.
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Open-label, Phase I Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis (ALS)
- ClinicalTrials.gov ID
- NCT06493279
- Acronym / sponsor ID
- RJK002-0001-R
- Conditions
- Amyotrophic Lateral Sclerosis Als
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 9 participants (estimated)
- Lead sponsor
- RJK Biopharma LtdIndustry (pharma, biotech, devices)
- Collaborators
- Peking University Third Hospital
- Study start
- 24 Sep 2024 (actual)
- Primary completion
- 30 Nov 2026 (estimated)
- Study completion
- 30 Nov 2030 (estimated)
- First posted
- 9 Jul 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 the study aims to evaluate the safety and tolerability of a single intrathecal injection of RJK002 in subjects with amyotrophic lateral sclerosis (ALS), and to determine the recommended Phase II dose (RP2D).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RJK002 Intrathecal injection | Experimental | Drug: RJK002 Intrathecal injectionEligible subjects will receive a single intrathecal administration of investigational product. 9 subjects will be involved in 3 doses cohort from low to high: 6E13 vg/person, 1.2E14 vg/person, and 2.4E14 vg/person, 3 subjects for each cohort. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
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 3E13vg (3ml), 6E13vg(6ml), and 1.2E14 vg(12ml) per 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 after administration
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 20 Nov 2025.
- Individual participant dataSharing planned: Yes. Part of the data are planed to share on Oct 2025, Oct 2026, and Sep 2032.
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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.
Dongsheng Fan, PhD principal investigator
Peking University Third Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing RJK002 Intrathecal injection in people with Amyotrophic Lateral Sclerosis Als, 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?
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 20 Nov 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?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 20 Nov 2025.