Clinical Trial: Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
Study Status: Completed
Recruit Status: Completed
Study Type: Observational
Official Title: Long-Term Follow-Up Safety and Preliminary Efficacy Study of Human Central Nervous System Stem Cell (HuCNS-SC®) Transplantation in Subjects With Connatal Pelizaeus-Merzbache
Brief Summary: The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).
Detailed Summary:
Only subjects who underwent HuCNS-SC transplantation under Protocol CL-N01-PMD will be enrolled in this long term follow-up study.
Subjects will return to the site six months and one year after completion of the Phase I study and then annually for a total study duration of four years. Phone calls will also be made by the Investigator to the subject's parent/legal guardian bi-annually to conduct a phone visit through the four-year duration of the study.
Sponsor: StemCells, Inc.
Current Primary Outcome: Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs. [ Time Frame: 4 years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- Preliminary efficacy using Bayley-III and Callier-Azusa Scale. [ Time Frame: 4 years ]Changes compared to baseline
- Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP). [ Time Frame: 4 years ]Changes compared to baseline
Original Secondary Outcome: Same as current
Information By: StemCells, Inc.
Dates:
Date Received: July 6, 2011
Date Started: June 2011
Date Completion:
Last Updated: May 11, 2016
Last Verified: May 2016
