Clinical Trial: Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
Study Status: Completed
Recruit Status: Completed
Study Type: Interventional
Official Title: Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)
Brief Summary: The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).
Detailed Summary: Enrolled subjects will be transplanted with HuCNS-SC cells into the brain and will receive immunosuppression for nine months. The study observation period is for one year after transplant surgery. Thereafter, subjects will be enrolled in a long-term observational follow-up study for four years.
Sponsor: StemCells, Inc.
Current Primary Outcome: Safety assessment through clinical neurological and MRI evaluation. [ Time Frame: one year post transplant ]
Original Primary Outcome: Same as current
Current Secondary Outcome: MRI examination for post-transplant myelination [ Time Frame: one year post transplant ]
Original Secondary Outcome: Same as current
Information By: StemCells, Inc.
Dates:
Date Received: October 28, 2009
Date Started: November 2009
Date Completion:
Last Updated: January 13, 2015
Last Verified: January 2015