NCT01005004

Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: StemCells, Inc. · Industry
• Sex: Male
• Age: 6 Months – 5 Years
• Healthy volunteers: Not accepted

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 description

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.

Conditions

• Pelizaeus-Merzbacher Disease

Interventions

Type	Name	Description
BIOLOGICAL	HuCNS-SC cells	intracerebral transplantation

Timeline

• Start date: 2009-11-01
• Primary completion: 2012-10-01
• Completion: 2012-12-01
• First posted: 2009-10-30
• Last updated: 2015-01-15

Locations

1 site across 1 country: United States