Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
The purpose of this Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis. The study will also measure post-transplantation disease progression.
|Study Design:||Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Nervous System Stem Cell Transplantation in Subjects With Non-Refractory Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis|
- number of participants with adverse events. [ Time Frame: one year following transplantation ] [ Designated as safety issue: Yes ]Safety will be measured by number of adverse events and changes in neurological status.
- Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS [ Time Frame: one year following transplantation ] [ Designated as safety issue: No ]
|Study Start Date:||November 2010|
|Study Completion Date:||April 2011|
|Primary Completion Date:||April 2011 (Final data collection date for primary outcome measure)|
Surgery to implant human CNS stem cells
Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress immune system and a series of follow-up assessments.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01238315
|United States, Oregon|
|Oregon Health and Science University|
|Portland, Oregon, United States, 97239|
|Principal Investigator:||Nathan Selden, MD, PhD||Oregon Health and Science University|