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

Completed

• Conditions: Pelizaeus-Merzbacher Disease; PMD
• Interventions: Biological: HuCNS-SC transplant in the lead-in phase

• Registration Number: NCT01391637
• Lead Sponsor: StemCells, Inc.

Brief Summary

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).

Detailed Description

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 Timeline

• Study Start: 2011-06
• Study First Submitted: 2011-07-06
• Study First Submitted QC: 2011-07-08
• Study First Posted: 2011-07-12
• Primary Completion: 2016-03
• Study Completion: 2016-03
• Status Verified: 2016-05
• Last Update Submitted: 2016-05-11
• Last Update Posted: 2016-05-13

Recruitment & Eligibility

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

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.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
HuCNS-SC transplanted subjects in the lead-in phase	HuCNS-SC transplant in the lead-in phase	Subjects who had HuCNS-SC transplant in the lead-in phase study CL-N01-PMD

Primary Outcome Measures

Name	Time	Method
Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.	4 years

Secondary Outcome Measures

Name	Time	Method
Preliminary efficacy using Bayley-III and Callier-Azusa Scale.	4 years	Changes compared to baseline
Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP).	4 years	Changes compared to baseline

Trial Locations

Locations (1)

UCSF Medical Center; 🇺🇸 San Francisco, California, United States