Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT01005004 |
Recruitment Status :
Completed
First Posted : October 30, 2009
Last Update Posted : January 15, 2015
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Pelizaeus-Merzbacher Disease | Biological: HuCNS-SC cells | Phase 1 |
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 |

Arm | Intervention/treatment |
---|---|
Experimental: HuCNS-SC cells
Intracerebral implantation of HuCNS-SC via direct injection during surgery
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Biological: HuCNS-SC cells
intracerebral transplantation
Other Name: Human central nervous system stem cells |
- Safety assessment through clinical neurological and MRI evaluation. [ Time Frame: one year post transplant ]
- MRI examination for post-transplant myelination [ Time Frame: one year post transplant ]

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Ages Eligible for Study: | 6 Months to 5 Years (Child) |
Sexes Eligible for Study: | Male |
Accepts 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

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
United States, California | |
University of California, San Francisco | |
San Francisco, California, United States, 94143 |
Study Director: | Stephen Huhn, MD | StemCells, Inc. |
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 |
PMD stem cells Pelizaeus-Merzbacher Disease human stem cells central nervous system stem cells |
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 |