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

NCT ID: NCT01005004

Last Updated: 2015-01-15

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

Clinical Phase

PHASE1

Total Enrollment

4 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-11-30

Study Completion Date

2012-12-31

Brief Summary

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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).

Detailed Description

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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.

Conditions

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Pelizaeus-Merzbacher Disease

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Blinding Strategy

NONE

Study Groups

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HuCNS-SC cells

Intracerebral implantation of HuCNS-SC via direct injection during surgery

Group Type EXPERIMENTAL

HuCNS-SC cells

Intervention Type BIOLOGICAL

intracerebral transplantation

Interventions

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HuCNS-SC cells

intracerebral transplantation

Intervention Type BIOLOGICAL

Other Intervention Names

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Human central nervous system stem cells

Eligibility Criteria

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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
Minimum Eligible Age

6 Months

Maximum Eligible Age

5 Years

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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University of California, San Francisco

San Francisco, California, United States

Site Status

Countries

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United States

Other Identifiers

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CL-N01-PMD

Identifier Type: -

Identifier Source: org_study_id

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