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

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

Genetics Home Reference related topics: Pelizaeus-Merzbacher disease RNAse T2-deficient leukoencephalopathy Leukoencephalopathy with vanishing white matter Megalencephalic leukoencephalopathy with subcortical cysts

Genetic and Rare Diseases Information Center resources: Pelizaeus-Merzbacher Disease

U.S. FDA Resources

Groups and Cohorts

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

Primary Outcome Measures :

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

Secondary Outcome Measures :

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

Information from the National Library of Medicine

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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

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

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United States, California
UCSF Medical Center
San Francisco, California, United States, 94143

Sponsors and Collaborators

StemCells, Inc.

Investigators

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Study Director:	Stephen Huhn, MD	StemCells, Inc.

More Information

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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:

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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

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