NCT06454682

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

A Single-site, Single-arm, Open-label, IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of AAV-RJK002 in Patients With Amyotrophic Lateral Sclerosis (ALS)

• Status: Active Not Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 9 (estimated)
• Sponsor: RJK Biopharma Ltd · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	RJK002 Intrathecal injection	Eligible subjects will receive a single intrathecal administration of investigational product with dose 3E13 vg/person, 6E13 vg/person, and 1.2E14 vg/person sequentially.

Timeline

• Start date: 2023-09-11
• Primary completion: 2025-06-21
• Completion: 2031-08-02
• First posted: 2024-06-12
• Last updated: 2025-08-28

Locations

1 site across 1 country: China