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

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ClinicalTrials.gov Identifier: NCT01005004
Recruitment Status : Completed
First Posted : October 30, 2009
Last Update Posted : January 15, 2015
Sponsor:
Information provided by (Responsible Party):
StemCells, Inc.

Study Description
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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).

Condition or disease Intervention/treatment Phase
Pelizaeus-Merzbacher Disease Biological: HuCNS-SC cells Phase 1

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.
Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Official Title: Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)
Study Start Date : November 2009
Actual Primary Completion Date : October 2012
Actual Study Completion Date : December 2012

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Arms and Interventions
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Arm Intervention/treatment
Experimental: HuCNS-SC cells
Intracerebral implantation of HuCNS-SC via direct injection during surgery
 Biological: HuCNS-SC cells
intracerebral transplantation
Other Name: Human central nervous system stem cells


Outcome Measures
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Primary Outcome Measures :
  1. Safety assessment through clinical neurological and MRI evaluation. [ Time Frame: one year post transplant ]

Secondary Outcome Measures :
  1. MRI examination for post-transplant myelination [ Time Frame: one year post transplant ]

Eligibility Criteria
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Ages Eligible for Study:   6 Months to 5 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01005004


Locations
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United States, California
University of California, San Francisco
San Francisco, California, United States, 94143
Sponsors and Collaborators
StemCells, Inc.
Investigators
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Study Director: Stephen Huhn, MD StemCells, Inc.
More Information
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Responsible Party: StemCells, Inc.
ClinicalTrials.gov Identifier: NCT01005004    
Other Study ID Numbers: CL-N01-PMD
First Posted: October 30, 2009    Key Record Dates
Last Update Posted: January 15, 2015
Last Verified: January 2015
Keywords provided by StemCells, Inc.:
PMD
stem cells
Pelizaeus-Merzbacher Disease
human stem cells
central nervous system stem cells
Additional relevant MeSH terms:
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Pelizaeus-Merzbacher Disease
Hereditary Central Nervous System Demyelinating Diseases
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
 Leukoencephalopathies
Demyelinating Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases