Human Neural Progenitor Cells Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis
Study Purpose:
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.
Disease:
Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALSStudy Type:
Interventional TrialStudy Category:
Stem CellStudy Status:
Not enrollingPhase:
Phase IStudy Chair(s)/Principal Investigator(s):
Robert H. Baloh, MD, PhD, Cedars-Sinai Medical Center
Clinicaltrials.gov ID (11 digit #):
NCT02943850Neals Affiliated?
YesCoordinating Center Contact Information
Cedars-Sinai Medical CenterPeggy Allred / .(JavaScript must be enabled to view this email address) / 310-423-1791
.(JavaScript must be enabled to view this email address) Los Angeles, California 90048 United States
Full Study Summary:
This study will be the first to use a genetically modified progenitor cells to treat a neurodegenerative disease. This is a Phase 1/2a, 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)