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

StemCells, Inc

Status: Completed

Conditions: Pelizaeus-Merzbacher Disease; PMD

Treatments: Biological: HuCNS-SC transplant in the lead-in phase

Study type: Observational

Funder types: Industry

Identifiers

NCT01391637

CL-N02-PMD

Details and patient eligibility

About

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).

Full 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.

Enrollment: 4 patients

Sex: Male

Volunteers: No Healthy Volunteers

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.

Trial design

4 participants in 1 patient group

HuCNS-SC transplanted subjects in the lead-in phase

Description:

Subjects who had HuCNS-SC transplant in the lead-in phase study CL-N01-PMD

Treatment:

Biological: HuCNS-SC transplant in the lead-in phase