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

Completed · Phase 1

Conditions studied: Pelizaeus-Merzbacher Disease

In brief

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).

Key facts

Study ID

NCT01005004

Run by

StemCells, Inc.

People needed

4

Starts

2009-11-01

Expected to finish

2012-12-01

Last updated by the study team

2015-01-15

Who can join

Age: 1 and older, up to 5. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• University of California, San Francisco — San Francisco, California, United States

Full record on ClinicalTrials.gov

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