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

This study has been completed.
Information provided by (Responsible Party):
StemCells, Inc. Identifier:
First received: July 6, 2011
Last updated: May 11, 2016
Last verified: May 2016
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 Intervention Phase
Pelizaeus-Merzbacher Disease PMD Biological: HuCNS-SC transplant in the lead-in phase Phase 1

Study Type: Observational
Study Design: 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)

Resource links provided by NLM:

Further study details as provided by StemCells, Inc.:

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

Enrollment: 4
Study Start Date: June 2011
Study Completion Date: March 2016
Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
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

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.


Ages Eligible for Study:   Child, Adult, Senior
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01391637

United States, California
UCSF Medical Center
San Francisco, California, United States, 94143
Sponsors and Collaborators
StemCells, Inc.
Study Director: Stephen Huhn, MD StemCells, Inc.
  More Information

Responsible Party: StemCells, Inc. Identifier: NCT01391637     History of Changes
Other Study ID Numbers: CL-N02-PMD
Study First Received: July 6, 2011
Last Updated: May 11, 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
Demyelinating Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases processed this record on September 21, 2017