Pelizaeus-Merzbacher Disease Clinical Trial
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-Merzbacher Disease (PMD)
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).
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.
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Observational Model: Cohort, Time Perspective: Prospective
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