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

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: NCT01005004
Recruitment Status : Completed
First Posted : October 30, 2009
Last Update Posted : January 15, 2015
Sponsor:
Information provided by (Responsible Party):
StemCells, Inc.

Tracking Information
First Submitted Date  ICMJE October 28, 2009
First Posted Date  ICMJE October 30, 2009
Last Update Posted Date January 15, 2015
Study Start Date  ICMJE November 2009
Actual Primary Completion Date October 2012   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 28, 2009)
Safety assessment through clinical neurological and MRI evaluation. [ Time Frame: one year post transplant ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 28, 2009)
MRI examination for post-transplant myelination [ Time Frame: one year post transplant ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
Official Title  ICMJE Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)
Brief Summary 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).
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Condition  ICMJE Pelizaeus-Merzbacher Disease
Intervention  ICMJE Biological: HuCNS-SC cells
intracerebral transplantation
Other Name: Human central nervous system stem cells
Study Arms  ICMJE Experimental: HuCNS-SC cells
Intracerebral implantation of HuCNS-SC via direct injection during surgery
Intervention: Biological: HuCNS-SC cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: October 28, 2009)
4
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE December 2012
Actual Primary Completion Date October 2012   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 6 Months to 5 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01005004
Other Study ID Numbers  ICMJE CL-N01-PMD
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party StemCells, Inc.
Study Sponsor  ICMJE StemCells, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Stephen Huhn, MD StemCells, Inc.
PRS Account StemCells, Inc.
Verification Date January 2015

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP