An IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With ALS

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.

Timeline

Start

2023-09-11

Primary completion

2025-06-21

Completion

2031-08-02

Drugs

Indications