Trial record 2 of 3 for:    "Pelizaeus-Merzbacher disease"

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

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
StemCells, Inc. Identifier:
First received: July 6, 2011
Last updated: November 7, 2013
Last verified: November 2013

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
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 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Preliminary efficacy using Bayley-III and Callier-Azusa Scale. [ Time Frame: 4 years ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    Changes compared to baseline

Estimated Enrollment: 4
Study Start Date: June 2011
Estimated Study Completion Date: June 2016
Estimated Primary Completion Date: February 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.


Genders 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
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

No publications provided

Responsible Party: StemCells, Inc. Identifier: NCT01391637     History of Changes
Other Study ID Numbers: CL-N02-PMD
Study First Received: July 6, 2011
Last Updated: November 7, 2013
Health Authority: United States: Food and Drug Administration

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 April 16, 2014