Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
NCT ID: NCT01005004
Last Updated: 2015-01-15
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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COMPLETED
PHASE1
4 participants
INTERVENTIONAL
2009-11-30
2012-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
NONE
Study Groups
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HuCNS-SC cells
Intracerebral implantation of HuCNS-SC via direct injection during surgery
HuCNS-SC cells
intracerebral transplantation
Interventions
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HuCNS-SC cells
intracerebral transplantation
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene
* MRI consistent with PMD as interpreted by a qualified neuroradiologist
Exclusion Criteria
* 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
6 Months
5 Years
MALE
No
Sponsors
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StemCells, Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Stephen Huhn, MD
Role: STUDY_DIRECTOR
StemCells, Inc.
Locations
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University of California, San Francisco
San Francisco, California, United States
Countries
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Other Identifiers
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CL-N01-PMD
Identifier Type: -
Identifier Source: org_study_id
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