Ignite Creation Date:
2024-06-16 @ 11:51 AM
Last Modification Date:
2024-10-26 @ 3:31 PM
Study NCT ID:
NCT06454682
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-07-03
First Post:
2024-06-03
Brief Title:
An IIT Clinical Study to Evaluate the Safety and Efficacy of a Single Intrathecal Injection of RJK002 in Patients With Amyotrophic Lateral Sclerosis ALS
Sponsor:
Rejukon Biopharm Inc
Organization:
Rejukon Biopharm Inc
Study Overview
Official Title:
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:
NOT_YET_RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief 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 6E13 vgperson 6 mL 12E14 vgperson 12 mL and 24E14 vgperson 24 mL 3 subjects will be enrolled in each dose cohort The dose level will be escalated sequentially from low to high
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 vgperson 6 mL 12E14 vgperson 12 mL and 24E14 vgperson 24 mL 3 subjects will be enrolled in each dose cohort The dose level will be escalated sequentially from low to high
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None