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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

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