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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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23 May 2016 |
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Main ID: |
NCT01391637 |
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Date of registration:
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06/07/2011 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
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Scientific title:
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Long-Term Follow-Up Safety and Preliminary Efficacy Study of Human Central Nervous System Stem Cell (HuCNS-SC®) Transplantation in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD) |
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Date of first enrolment:
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June 2011 |
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Target sample size:
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4 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT01391637 |
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Study type:
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Observational |
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Study design:
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Observational Model: Cohort, Time Perspective: Prospective
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Phase:
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Countries of recruitment
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United States
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Contacts
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Name:
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Stephen Huhn, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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StemCells, Inc. |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Subjects who received HuCNS-SC cells under Protocol CL-N01-PMD
Exclusion Criteria:
- Subjects who received off-protocol immunosuppressive medications.
- Subjects who are concurrently enrolled in another investigational study.
Age minimum:
N/A
Age maximum:
N/A
Gender:
Male
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Health Condition(s) or Problem(s) studied
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Pelizaeus-Merzbacher Disease
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PMD
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Intervention(s)
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Biological: HuCNS-SC transplant in the lead-in phase
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Primary Outcome(s)
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Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.
[Time Frame: 4 years]
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Secondary Outcome(s)
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Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP).
[Time Frame: 4 years]
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Preliminary efficacy using Bayley-III and Callier-Azusa Scale.
[Time Frame: 4 years]
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Secondary ID(s)
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CL-N02-PMD
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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