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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
StemCells, Inc.
ClinicalTrials.gov Identifier:
NCT01005004
First received: October 28, 2009
Last updated: April 23, 2013
Last verified: April 2013
History of Changes

October 28, 2009
April 23, 2013
November 2009
October 2012   (final data collection date for primary outcome measure)
Safety assessment through clinical neurological and MRI evaluation. [ Time Frame: one year post transplant ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT01005004 on ClinicalTrials.gov Archive Site
MRI examination for post-transplant myelination [ Time Frame: one year post transplant ] [ Designated as safety issue: No ]
Same as current
Not Provided
Not Provided
 
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)

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).

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.
Interventional
Phase 1
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Pelizaeus-Merzbacher Disease
Biological: HuCNS-SC cells
intracerebral transplantation
Other Name: Human central nervous system stem cells
Experimental: HuCNS-SC cells
Intracerebral implantation of HuCNS-SC via direct injection during surgery
Intervention: Biological: HuCNS-SC cells
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
4
December 2012
October 2012   (final data collection date for primary outcome measure)

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
Male
6 Months to 5 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01005004
CL-N01-PMD
Yes
StemCells, Inc.
StemCells, Inc.
Not Provided
Study Director: Stephen Huhn, MD StemCells, Inc.
StemCells, Inc.
April 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP