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

StemCells, Inc.4 enrolled

Overview

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).

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

Allocation

Masking

NONE

Enrollment

Conditions

Pelizaeus-Merzbacher Disease

Interventions

HuCNS-SC cellsBIOLOGICAL

intracerebral transplantation

Eligibility

Sex: MALEMin age: 6 MonthsMax age: 5 Years

Medical Language ↔ Plain English

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

Locations (1)

University of California, San Francisco

San Francisco, California, United States

Outcomes

Primary Outcomes

Safety assessment through clinical neurological and MRI evaluation.

Time frame: one year post transplant

Secondary Outcomes

MRI examination for post-transplant myelination

Time frame: one year post transplant

Data from ClinicalTrials.gov

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