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Completed

NCT Number: NCT01391637

Long-Term Follow-Up Study of Human Stem Cells Transplanted 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).

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Key information

Conditions

Pelizaeus-Merzbacher Disease Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Demyelinating Diseases Genetic Diseases, Inborn Genetic Diseases, X-Linked Hereditary Central Nervous System Demyelinating Diseases Leukoencephalopathies Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Nutritional and Metabolic Diseases PMD

Sex eligibility

Male

Study type

Observational

Primary location

UCSF Medical Center

San Francisco, California, 94143, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

HuCNS-SC transplant in the lead-in phase

Biological

Long-term safety follow-up study

Primary outcomes

  1. Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.

    Time frame: 4 years

Secondary outcomes

  1. Preliminary efficacy using Bayley-III and Callier-Azusa Scale.

    Time frame: 4 years

    Changes compared to baseline

  2. 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

Sponsors and collaborators

Lead sponsor

StemCells, Inc.

Industry

Registry information

Official study title

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)

Important dates

Study start
2011
Primary completion
2016
Study completion
2016
First posted
Jul 12, 2011
Registry last updated
May 13, 2016

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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