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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
StemCells, Inc.
ClinicalTrials.gov Identifier:
NCT01005004
First received: October 28, 2009
Last updated: April 23, 2013
Last verified: April 2013
  Purpose

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.

  Eligibility

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

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
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 ClinicalTrials.gov identifier: NCT01005004

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

Additional Information:
No publications provided

Responsible Party: StemCells, Inc.
ClinicalTrials.gov 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.:
PMD
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
Leukoencephalopathies
Demyelinating Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Metabolic Diseases

ClinicalTrials.gov processed this record on September 22, 2014