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

May 11, 2016 updated by: StemCells, Inc.

Long-Term Follow-Up Safety and Preliminary Efficacy Study of Human Central Nervous System Stem Cell (HuCNS-SC®) Transplantation in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)

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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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 Type

Observational

Enrollment (Actual)

4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • California
      • San Francisco, California, United States, 94143
        • UCSF Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Non-Probability Sample

Study Population

PMD Subjects who underwent transplantation of HuCNS-SC cells under CL-N01-PMD study

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
HuCNS-SC transplanted subjects in the lead-in phase
Subjects who had HuCNS-SC transplant in the lead-in phase study CL-N01-PMD
Biological: HuCNS-SC transplant in the lead-in phase
Long-term safety follow-up study

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.
Time Frame: 4 years
4 years

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

StemCells, Inc.

Investigators

  • Study Director: Stephen Huhn, MD, StemCells, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2011

Primary Completion (Actual)

March 1, 2016

Study Completion (Actual)

March 1, 2016

Study Registration Dates

First Submitted

July 6, 2011

First Submitted That Met QC Criteria

July 8, 2011

First Posted (Estimate)

July 12, 2011

Study Record Updates

Last Update Posted (Estimate)

May 13, 2016

Last Update Submitted That Met QC Criteria

May 11, 2016

Last Verified

May 1, 2016

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CL-N02-PMD

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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