Pelizaeus-Merzbacher Disease, PMD
Conditions
Keywords
Long term follow-up, HuCNS-SC cells, human central nervous system stem cells
Brief 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.
Interventions
BIOLOGICALHuCNS-SC transplant in the lead-in phase
Long-term safety follow-up study
Sponsors
StemCells, Inc.
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
Inclusion criteria
* Subjects who received HuCNS-SC cells under Protocol CL-N01-PMD
Exclusion criteria
* Subjects who received off-protocol immunosuppressive medications. * Subjects who are concurrently enrolled in another investigational study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs. | 4 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Preliminary efficacy using Bayley-III and Callier-Azusa Scale. | 4 years | Changes compared to baseline |
| Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP). | 4 years | Changes compared to baseline |
Countries
United States
Outcome results
None listed