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

Phase 1

Completed

• Conditions: Pelizaeus-Merzbacher Disease
• Interventions: Biological: HuCNS-SC cells

• Registration Number: NCT01005004
• Lead Sponsor: StemCells, Inc.

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 Timeline

• Study First Submitted: 2009-10-28
• Study First Submitted QC: 2009-10-28
• Study First Posted: 2009-10-30
• Study Start: 2009-11
• Primary Completion: 2012-10
• Study Completion: 2012-12
• Status Verified: 2015-01
• Last Update Submitted: 2015-01-13
• Last Update Posted: 2015-01-15

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 4

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

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
HuCNS-SC cells	HuCNS-SC cells	Intracerebral implantation of HuCNS-SC via direct injection during surgery

Primary Outcome Measures

Name	Time	Method
Safety assessment through clinical neurological and MRI evaluation.	one year post transplant

Secondary Outcome Measures

Name	Time	Method
MRI examination for post-transplant myelination	one year post transplant

Trial Locations

Locations (1)

University of California, San Francisco; 🇺🇸 San Francisco, California, United States