Ignite Creation Date:
2024-05-06 @ 9:16 AM
Last Modification Date:
2024-10-26 @ 12:12 PM
Study NCT ID:
NCT02943850
Status:
COMPLETED
Last Update Posted:
2020-07-15
First Post:
2016-10-20
Brief Title:
CNS10-NPC-GDNF for the Treatment of 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 for the Treatment of Amyotrophic Lateral Sclerosis
Status:
COMPLETED
Status Verified Date:
2020-07
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 spinal cord 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 neuronal 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 neuronal cells Therefore the cells are called CNS10-NPC-GDNF The investigational treatment has been tested in animals but it has not yet been tested in people In this study we want to learn if CNS10-NPC-GDNF cells are safe to transplant into the spinal cords of people
Detailed Description:
This study will be the first to use a genetically modified progenitor cells to treat a neurodegenerative disease This is a Phase 12a single-center blinded as to side of injection safety study of two escalating doses of human neural progenitor cells expressing GDNF CNS10-NPC-GDNF delivered unilaterally to the lumbar region in ALS subjects with moderate leg involvement
Subjects meeting all Eligibility Criteria and providing Informed Consent will be enrolled in one of two 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 two weeks between surgeries
Specific aims
Safety as evaluated by
Adverse Events and Serious Adverse Events
Clinical laboratory assessments as clinically indicated hematology chemistry immunology
Subjects meeting all Eligibility Criteria and providing Informed Consent will be enrolled in one of two 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 two weeks between surgeries
Specific aims
Safety as evaluated by
Adverse Events and Serious Adverse Events
Clinical laboratory assessments as clinically indicated hematology chemistry immunology
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
True
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None