Ignite Creation Date:
2024-05-06 @ 5:25 PM
Last Modification Date:
2024-10-26 @ 2:28 PM
Study NCT ID:
NCT05306457
Status:
RECRUITING
Last Update Posted:
2024-02-06
First Post:
2021-12-21
Brief Title:
CNS10-NPC-GDNF Delivered to the Motor Cortex for ALS
Sponsor:
Cedars-Sinai Medical Center
Organization:
Cedars-Sinai Medical Center
Study Overview
Official Title:
Human Neural Progenitor Cells Secreting Glial Cell Line-Derived Neurotrophic Factor CNS10-NPC-GDNF Delivered to the Motor Cortex for the Treatment of Amyotrophic Lateral Sclerosis
Status:
RECRUITING
Status Verified Date:
2024-02
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 investigator is examining the safety of transplanting cells that have been engineered to produce a growth factor into the motor cortex brain of patients with Amyotrophic Lateral Sclerosis ALS The cells are called neural progenitor cells which are a type of stem cell that can become several different types of cells in the nervous system These cells have been derived to specifically become astrocytes which is a type of neural cell The growth factor is called glial cell line-derived neurotrophic factor or GDNF GDNF is a protein that promotes the survival of many types of neural cells Therefore the cells are called CNS10-NPC-GDNF The investigational treatment has been tested in people by delivering it to the spinal cord However it has only been delivered to the motor cortex of animals In this study we want to learn if CNS10-NPC-GDNF cells are safe to transplant into the motor cortex brain of people
Detailed Description:
This study will be the first to use a genetically modified progenitor cell line delivered to the motor cortex to treat a neurodegenerative disease This is a Phase 12a single-center safety study of two escalating doses of human neural progenitor cells expressing GDNF CNS10-NPC-GDNF delivered unilaterally to the hand-knob area of the motor cortex of patients with ALS
Subjects meeting all Eligibility Criteria and providing Informed Consent will be enrolled in one of three sequential dosing groups Subjects will be treated sequentially with a minimum of one month interval between surgeries for the first three subjects in each dosing cohort The remaining subjects in the cohort will be treated with a minimum interval of at least one week between surgeries
Primary Outcome
Safety as evaluated by
Adverse Events and Serious Adverse Events
Post-op MRI andor CT with contrast and as clinically indicated
Subjects meeting all Eligibility Criteria and providing Informed Consent will be enrolled in one of three sequential dosing groups Subjects will be treated sequentially with a minimum of one month interval between surgeries for the first three subjects in each dosing cohort The remaining subjects in the cohort will be treated with a minimum interval of at least one week between surgeries
Primary Outcome
Safety as evaluated by
Adverse Events and Serious Adverse Events
Post-op MRI andor CT with contrast and as clinically indicated
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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