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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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ClinicalTrials.gov Identifier: NCT01391637
Recruitment Status : Completed
First Posted : July 12, 2011
Last Update Posted : May 13, 2016
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 long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).

Condition or disease Intervention/treatment
Pelizaeus-Merzbacher Disease PMD Biological: HuCNS-SC transplant in the lead-in phase

Detailed Description:

Only subjects who underwent HuCNS-SC transplantation under Protocol CL-N01-PMD will be enrolled in this long term follow-up study.

Subjects will return to the site six months and one year after completion of the Phase I study and then annually for a total study duration of four years. Phone calls will also be made by the Investigator to the subject's parent/legal guardian bi-annually to conduct a phone visit through the four-year duration of the study.


Study Design
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Study Type : Observational
Actual Enrollment : 4 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: 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)
Study Start Date : June 2011
Actual Primary Completion Date : March 2016
Actual Study Completion Date : March 2016

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort Intervention/treatment
HuCNS-SC transplanted subjects in the lead-in phase
Subjects who had HuCNS-SC transplant in the lead-in phase study CL-N01-PMD
 Biological: HuCNS-SC transplant in the lead-in phase
Long-term safety follow-up study


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs. [ Time Frame: 4 years ]

Secondary Outcome Measures :
  1. Preliminary efficacy using Bayley-III and Callier-Azusa Scale. [ Time Frame: 4 years ]
    Changes compared to baseline

  2. Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP). [ Time Frame: 4 years ]
    Changes compared to baseline


Eligibility Criteria
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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
PMD Subjects who underwent transplantation of HuCNS-SC cells under CL-N01-PMD study
Criteria

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.
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): NCT01391637


Locations
United States, California
UCSF Medical Center
San Francisco, California, United States, 94143
Sponsors and Collaborators
StemCells, Inc.
Investigators
Study Director: Stephen Huhn, MD StemCells, Inc.
More Information
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information

Responsible Party: StemCells, Inc.
ClinicalTrials.gov Identifier: NCT01391637     History of Changes
Other Study ID Numbers: CL-N02-PMD
First Posted: July 12, 2011    Key Record Dates
Last Update Posted: May 13, 2016
Last Verified: May 2016

Keywords provided by StemCells, Inc.:
Long term follow-up
HuCNS-SC cells
human central nervous system stem cells

Additional relevant MeSH terms:
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