Pelizaeus-Merzbacher Disease
Conditions
Keywords
PMD, stem cells, Pelizaeus-Merzbacher Disease, human stem cells, central nervous system stem cells
Brief 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.
Interventions
BIOLOGICALHuCNS-SC cells
intracerebral transplantation
Sponsors
StemCells, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Masking
NONE
Eligibility
Sex/Gender
MALE
Age
6 Months to 5 Years
Healthy volunteers
No
Inclusion criteria
* Confirmed clinical diagnosis of connatal PMD * Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene * MRI consistent with PMD as interpreted by a qualified neuroradiologist
Exclusion criteria
* Other significant congenital brain abnormality not related to PMD * Previous participation in gene transfer or cell transplant trial * Presence of neurological signs and symptoms not consistent with PMD * Current or prior malignancy * Prior organ, tissue or bone marrow transplant
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety assessment through clinical neurological and MRI evaluation. | one year post transplant |
Secondary
| Measure | Time frame |
|---|---|
| MRI examination for post-transplant myelination | one year post transplant |
Countries
United States
Outcome results
None listed