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Clinical Trials associated with RJK-002A Single-arm, Open-label, Phase I Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis (ALS)
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 Phase II dose (RP2D).
/ Active, not recruitingEarly Phase 1 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)
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: 6E13 vg/person (6 mL), 1.2E14 vg/person (12 mL), and 2.4E14 vg/person (24 mL). 3 subjects will be enrolled in each dose cohort. The dose level will be escalated sequentially from low to high.
Start Date01 Nov 2023 |
Sponsor / Collaborator- |
100 Clinical Results associated with RJK-002
100 Translational Medicine associated with RJK-002
100 Patents (Medical) associated with RJK-002
100 Deals associated with RJK-002