An IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJ… (NCT06454682) | Clinical Trial Compass
Active — Not RecruitingEarly Phase 1
An IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With ALS
China9 participantsStarted 2023-09-11
Plain-language 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 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.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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 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;
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
incidence of adverse events (AEs)/serious adverse events (SAEs)
Timeframe: Each visit within 5 years after administration
. 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).
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;
. 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;
. 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;