Evaluate the Safety and Efficacy of Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis

Launched by RJK BIOPHARMA LTD · Jul 2, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a new treatment called RJK002 for patients with amyotrophic lateral sclerosis (ALS), a serious disease that affects the nerves and muscles. The main goal is to see if a single injection of RJK002 is safe and how well it works. Researchers want to find out the best dose to use in future studies. If you are between 18 and 74 years old, have been diagnosed with ALS within the last two years, and meet other health criteria, you might be eligible to participate.

Participants in this trial can expect to receive an injection of RJK002 and will be monitored closely for any side effects or changes in their condition. It's important to know that this is an early-phase study, meaning it’s one of the first steps in testing this treatment. The trial is currently looking for volunteers, and if you choose to participate, you will help researchers understand more about this potential new therapy for ALS.

Gender

ALL

Eligibility criteria

• Main Inclusion Criteria:
• • 1. Female or male subjects who are ≥ 18 years of age at screening;
• • 2. 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);
• • 3. The duration of the disease from the first symptom (any ALS symptom) prior to the screening visit must be less than 2 years (inclusive);
• • 4. 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;
• • 5. The forced vital capacity (FVC) of predicted during the screening period is ≥70% at screening;
• • 6. Body mass index (BMI) greater than 18 kg/m2 at screening;
• Main Exclusion Criteria:
• • 1. 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;
• • 2. 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);
• • 3. Subjects who refuse to take food and medication by nasal feeding tube during the study period due to swallowing dysfunction;