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

This study has been completed.

Sponsor:

ClinicalTrials.gov Identifier:

NCT01005004

First Posted: October 30, 2009

Last Update Posted: January 15, 2015

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.

Information provided by (Responsible Party):

StemCells, Inc.

Tracking Information

First Submitted Date ^ICMJE

October 28, 2009

First Posted Date ^ICMJE

October 30, 2009

Last Update Posted Date

January 15, 2015

Start Date ^ICMJE

November 2009

Primary Completion Date

October 2012 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Safety assessment through clinical neurological and MRI evaluation. [ Time Frame: one year post transplant ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT01005004 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

MRI examination for post-transplant myelination [ Time Frame: one year post transplant ]

Original Secondary Outcome Measures ^ICMJE

Same as current

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

Phase I Study of the Safety and Preliminary Efficacy of Intracerebral Transplantation of HuCNS-SC® Cells for Connatal Pelizaeus-Merzbacher Disease (PMD)

Brief Summary

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

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.

Study Type ^ICMJE

Interventional

Study Phase

Phase 1

Study Design ^ICMJE

Intervention Model: Single Group Assignment
Masking: None (Open Label)

Condition ^ICMJE

Pelizaeus-Merzbacher Disease

Intervention ^ICMJE

Biological: HuCNS-SC cells

intracerebral transplantation

Other Name: Human central nervous system stem cells

Study Arms

Experimental: HuCNS-SC cells

Intracerebral implantation of HuCNS-SC via direct injection during surgery

Intervention: Biological: HuCNS-SC cells

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

December 2012

Primary Completion Date

October 2012 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Sex/Gender

Sexes Eligible for Study:

Male

Ages

6 Months to 5 Years (Child)

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Listed Location Countries ^ICMJE

United States

Removed Location Countries

Administrative Information

NCT Number ^ICMJE

NCT01005004

Other Study ID Numbers ^ICMJE

CL-N01-PMD

Has Data Monitoring Committee

Yes

U.S. FDA-regulated Product

Not Provided

IPD Sharing Statement

Not Provided

Responsible Party

StemCells, Inc.

Study Sponsor ^ICMJE

StemCells, Inc.

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Study Director:

Stephen Huhn, MD

StemCells, Inc.

PRS Account

StemCells, Inc.

Verification Date

January 2015

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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