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

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

Tracking Information

• First Submitted Date: October 28, 2009
• First Posted Date: October 30, 2009
• Last Update Posted Date: January 15, 2015
• Study Start Date: November 2009
• Actual Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: October 28, 2009): Safety assessment through clinical neurological and MRI evaluation. [ Time Frame: one year post transplant ]
• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures (submitted: October 28, 2009): MRI examination for post-transplant myelination [ Time Frame: one year post transplant ]
• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects
• Official Title: 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: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label)
• Condition: Pelizaeus-Merzbacher Disease

Intervention

Biological: HuCNS-SC cells

intracerebral transplantation

Other Name: Human central nervous system stem cells

Study Arms

Experimental: HuCNS-SC cells

Intracerebral implantation of HuCNS-SC via direct injection during surgery

Intervention: Biological: HuCNS-SC cells

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: October 28, 2009): 4
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: December 2012
• Actual Primary Completion Date: October 2012 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: Male

• Ages: 6 Months to 5 Years (Child)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT01005004
• Other Study ID Numbers: CL-N01-PMD
• Has Data Monitoring Committee: Yes
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Responsible Party: StemCells, Inc.
• Study Sponsor: StemCells, Inc.
• Collaborators: Not Provided

Investigators

• Study Director: Stephen Huhn, MD; StemCells, Inc.

• PRS Account: StemCells, Inc.
• Verification Date: January 2015