Clinical Trial: Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis

Study Status: Withdrawn
Recruit Status: Withdrawn
Study Type: Interventional

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

Brief Summary: 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.

Detailed Summary: 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.
Sponsor: StemCells, Inc.

Current Primary Outcome: number of participants with adverse events. [ Time Frame: one year following transplantation ]

Safety will be measured by number of adverse events and changes in neurological status.


Original Primary Outcome: Safety assessment through clinical, neurological and MRI evaluation. [ Time Frame: one year following transplantation ]

Current Secondary Outcome: Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS [ Time Frame: one year following transplantation ]

Original Secondary Outcome: Improvement in neurological, neuropsychological development, MRI and MRS [ Time Frame: one year following transplantation ]

Information By: StemCells, Inc.

Dates:
Date Received: November 5, 2010
Date Started: November 2010
Date Completion:
Last Updated: January 13, 2015
Last Verified: January 2015