Status:
ACTIVE_NOT_RECRUITING
Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis
Lead Sponsor:
RJK Biopharma Ltd
Collaborating Sponsors:
Peking University Third Hospital
Conditions:
Amyotrophic Lateral Sclerosis Als
Eligibility:
All Genders
18+ years
Phase:
PHASE1
Brief 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 Ph...
Eligibility Criteria
Inclusion
- Main
- 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
- 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;
Key Trial Info
Start Date :
September 24 2024
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
November 30 2030
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT06493279
Start Date
September 24 2024
End Date
November 30 2030
Last Update
November 20 2025
Active Locations (1)
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1
Peking University Third Hospital
Beijing, China