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Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)

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: NCT01391637
Recruitment Status : Completed
First Posted : July 12, 2011
Last Update Posted : May 13, 2016
Information provided by (Responsible Party):
StemCells, Inc.

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.

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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

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

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

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

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.

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

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United States, California
UCSF Medical Center
San Francisco, California, United States, 94143
Sponsors and Collaborators
StemCells, Inc.
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Study Director: Stephen Huhn, MD StemCells, Inc.
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Responsible Party: StemCells, Inc.
ClinicalTrials.gov Identifier: NCT01391637    
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
Demyelinating Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases