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
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Sponsor:
StemCells, Inc.
Information provided by (Responsible Party):
StemCells, Inc.
Tracking Information | ||||
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First Submitted Date ICMJE | October 28, 2009 | |||
First Posted Date ICMJE | October 30, 2009 | |||
Last Update Posted Date | January 15, 2015 | |||
Study Start Date ICMJE | November 2009 | |||
Actual Primary Completion Date | October 2012 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
Safety assessment through clinical neurological and MRI evaluation. [ Time Frame: one year post transplant ] | |||
Original Primary Outcome Measures ICMJE | Same as current | |||
Change History | ||||
Current Secondary Outcome Measures ICMJE |
MRI examination for post-transplant myelination [ Time Frame: one year post transplant ] | |||
Original Secondary Outcome Measures ICMJE | Same as current | |||
Current Other Pre-specified Outcome Measures | Not Provided | |||
Original Other Pre-specified Outcome Measures | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects | |||
Official Title ICMJE | Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD) | |||
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). | |||
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 Type ICMJE | Interventional | |||
Study Phase ICMJE | Phase 1 | |||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) |
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Condition ICMJE | Pelizaeus-Merzbacher Disease | |||
Intervention ICMJE | Biological: HuCNS-SC cells
intracerebral transplantation
Other Name: Human central nervous system stem cells
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Study Arms ICMJE | Experimental: HuCNS-SC cells
Intracerebral implantation of HuCNS-SC via direct injection during surgery
Intervention: Biological: HuCNS-SC cells
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Publications * | Not Provided | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Completed | |||
Actual Enrollment ICMJE |
4 | |||
Original Estimated Enrollment ICMJE | Same as current | |||
Actual Study Completion Date ICMJE | December 2012 | |||
Actual Primary Completion Date | October 2012 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion Criteria:
Exclusion Criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | 6 Months to 5 Years (Child) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT01005004 | |||
Other Study ID Numbers ICMJE | CL-N01-PMD | |||
Has Data Monitoring Committee | Yes | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE | Not Provided | |||
Responsible Party | StemCells, Inc. | |||
Study Sponsor ICMJE | StemCells, Inc. | |||
Collaborators ICMJE | Not Provided | |||
Investigators ICMJE |
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PRS Account | StemCells, Inc. | |||
Verification Date | January 2015 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |