Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)

StemCells, Inc.4 enrolled

Overview

The purpose of this study is to determine the long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).

Only subjects who underwent HuCNS-SC transplantation under Protocol CL-N01-PMD will be enrolled in this long term follow-up study. Subjects will return to the site six months and one year after completion of the Phase I study and then annually for a total study duration of four years. Phone calls will also be made by the Investigator to the subject's parent/legal guardian bi-annually to conduct a phone visit through the four-year duration of the study.

Study Type

OBSERVATIONAL

Enrollment

Conditions

Pelizaeus-Merzbacher Disease PMD

Interventions

HuCNS-SC transplant in the lead-in phaseBIOLOGICAL

Long-term safety follow-up study

Eligibility

Sex: MALE

Medical Language ↔ Plain English

Inclusion Criteria: * Subjects who received HuCNS-SC cells under Protocol CL-N01-PMD Exclusion Criteria: * Subjects who received off-protocol immunosuppressive medications. * Subjects who are concurrently enrolled in another investigational study.

Locations (1)

UCSF Medical Center

San Francisco, California, United States

Outcomes

Primary Outcomes

Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.

Time frame: 4 years

Secondary Outcomes

Preliminary efficacy using Bayley-III and Callier-Azusa Scale.

Changes compared to baseline

Time frame: 4 years

Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP).

Changes compared to baseline

Time frame: 4 years

Data from ClinicalTrials.gov

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