Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
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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. |
ClinicalTrials.gov Identifier: NCT01391637 |
Recruitment Status :
Completed
First Posted : July 12, 2011
Last Update Posted : May 13, 2016
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Condition or disease | Intervention/treatment |
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Pelizaeus-Merzbacher Disease PMD | Biological: HuCNS-SC transplant in the lead-in phase |
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 |
Actual Enrollment : | 4 participants |
Observational Model: | Cohort |
Time Perspective: | Prospective |
Official 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) |
Study Start Date : | June 2011 |
Actual Primary Completion Date : | March 2016 |
Actual Study Completion Date : | March 2016 |

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
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Biological: HuCNS-SC transplant in the lead-in phase
Long-term safety follow-up study |
- Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs. [ Time Frame: 4 years ]
- Preliminary efficacy using Bayley-III and Callier-Azusa Scale. [ Time Frame: 4 years ]Changes compared to baseline
- 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

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Ages Eligible for Study: | Child, Adult, Older Adult |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Sampling Method: | Non-Probability Sample |
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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01391637
United States, California | |
UCSF Medical Center | |
San Francisco, California, United States, 94143 |
Study Director: | Stephen Huhn, MD | StemCells, Inc. |
Responsible Party: | StemCells, Inc. |
ClinicalTrials.gov Identifier: | NCT01391637 |
Other Study ID Numbers: |
CL-N02-PMD |
First Posted: | July 12, 2011 Key Record Dates |
Last Update Posted: | May 13, 2016 |
Last Verified: | May 2016 |
Long term follow-up HuCNS-SC cells human central nervous system stem cells |
Pelizaeus-Merzbacher Disease Hereditary Central Nervous System Demyelinating Diseases Brain Diseases, Metabolic, Inborn Brain Diseases, Metabolic Brain Diseases Central Nervous System Diseases Nervous System Diseases |
Leukoencephalopathies Demyelinating Diseases Genetic Diseases, X-Linked Genetic Diseases, Inborn Metabolism, Inborn Errors Metabolic Diseases |