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

Pelizaeus-Merzbacher Disease Clinical Trial

Official title:

Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01005004
• Other study ID #: CL-N01-PMD
• Status: Completed
• Phase: Phase 1
• First received: October 28, 2009
• Last updated: January 13, 2015
• Start date: November 2009
• Est. completion date: December 2012

Study information

• Verified date: January 2015
• Source: StemCells, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: December 2012
• Est. primary completion date: October 2012
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 6 Months to 5 Years

Eligibility

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

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label

Related Conditions & MeSH terms

• Pelizaeus-Merzbacher Disease

Intervention

Biological:
• HuCNS-SC cells
intracerebral transplantation

Locations

Country	Name	City	State
United States	University of California, San Francisco	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
StemCells, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety assessment through clinical neurological and MRI evaluation.		one year post transplant	Yes
Secondary	MRI examination for post-transplant myelination		one year post transplant	No

External Links

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