Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

This study has been completed.
Information provided by (Responsible Party):
StemCells, Inc. Identifier:
First received: October 28, 2009
Last updated: April 23, 2013
Last verified: April 2013

The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients with Connatal Pelizaeus-Merzbacher Disease (PMD).

Condition Intervention Phase
Pelizaeus-Merzbacher Disease
Biological: HuCNS-SC cells
Phase 1

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Official Title: Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)

Resource links provided by NLM:

Further study details as provided by StemCells, Inc.:

Primary Outcome Measures:
  • Safety assessment through clinical neurological and MRI evaluation. [ Time Frame: one year post transplant ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • MRI examination for post-transplant myelination [ Time Frame: one year post transplant ] [ Designated as safety issue: No ]

Enrollment: 4
Study Start Date: November 2009
Study Completion Date: December 2012
Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HuCNS-SC cells
Intracerebral implantation of HuCNS-SC via direct injection during surgery
Biological: HuCNS-SC cells
intracerebral transplantation
Other Name: Human central nervous system stem cells

Detailed Description:

Enrolled subjects will be transplanted with HuCNS-SC cells into the brain and will receive immunosuppression for nine months. The study observation period is for one year after transplant surgery. Thereafter, subjects will be enrolled in a long-term observational follow-up study for four years.


Ages Eligible for Study:   6 Months to 5 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Confirmed clinical diagnosis of connatal PMD
  • Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene
  • MRI consistent with PMD as interpreted by a qualified neuroradiologist

Exclusion Criteria:

  • Other significant congenital brain abnormality not related to PMD
  • Previous participation in gene transfer or cell transplant trial
  • Presence of neurological signs and symptoms not consistent with PMD
  • Current or prior malignancy
  • Prior organ, tissue or bone marrow transplant
  Contacts and Locations
Please refer to this study by its identifier: NCT01005004

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

Additional Information:
No publications provided

Responsible Party: StemCells, Inc. Identifier: NCT01005004     History of Changes
Other Study ID Numbers: CL-N01-PMD
Study First Received: October 28, 2009
Last Updated: April 23, 2013
Health Authority: United States: Food and Drug Administration

Keywords provided by StemCells, Inc.:
stem cells
Pelizaeus-Merzbacher Disease
human stem cells
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 17, 2014