NCT01391637

Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)

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)

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 4 (actual)
• Sponsor: StemCells, Inc. · Industry
• Sex: Male
• Age: —
• Healthy volunteers: Not accepted

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 description

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.

Conditions

• Pelizaeus-Merzbacher Disease
• PMD

Interventions

Type	Name	Description
BIOLOGICAL	HuCNS-SC transplant in the lead-in phase	Long-term safety follow-up study

Timeline

• Start date: 2011-06-01
• Primary completion: 2016-03-01
• Completion: 2016-03-01
• First posted: 2011-07-12
• Last updated: 2016-05-13

Locations

1 site across 1 country: United States