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

NCT ID: NCT01391637

Last Updated: 2016-05-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

4 participants

Study Classification

OBSERVATIONAL

Study Start Date

2011-06-30

Study Completion Date

2016-03-31

Brief Summary

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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

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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.

Conditions

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Pelizaeus-Merzbacher Disease PMD

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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HuCNS-SC transplanted subjects in the lead-in phase

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

HuCNS-SC transplant in the lead-in phase

Intervention Type BIOLOGICAL

Long-term safety follow-up study

Interventions

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HuCNS-SC transplant in the lead-in phase

Long-term safety follow-up study

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.
Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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StemCells, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Stephen Huhn, MD

Role: STUDY_DIRECTOR

StemCells, Inc.

Locations

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UCSF Medical Center

San Francisco, California, United States

Site Status

Countries

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United States

Other Identifiers

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CL-N02-PMD

Identifier Type: -

Identifier Source: org_study_id

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