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

Purpose

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).

Condition	Intervention	Phase
Pelizaeus-Merzbacher Disease PMD	Biological: HuCNS-SC transplant in the lead-in phase	Phase 1


Study Type:	Observational
Study Design:	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)

Resource links provided by NLM:

Genetics Home Reference related topics: Pelizaeus-Merzbacher disease leukoencephalopathy with vanishing white matter megalencephalic leukoencephalopathy with subcortical cysts succinic semialdehyde dehydrogenase deficiency

Genetic and Rare Diseases Information Center resources: Pelizaeus-Merzbacher Disease

U.S. FDA Resources

Further study details as provided by StemCells, Inc.:

Primary Outcome Measures:

Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs. [ Time Frame: 4 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Preliminary efficacy using Bayley-III and Callier-Azusa Scale. [ Time Frame: 4 years ] [ Designated as safety issue: No ]
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 ] [ Designated as safety issue: No ]
Changes compared to baseline


Enrollment:	4
Study Start Date:	June 2011
Study Completion Date:	March 2016
Primary Completion Date:	March 2016 (Final data collection date for primary outcome measure)

Groups/Cohorts	Assigned Interventions
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

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.

Eligibility


Ages Eligible for Study:	Child, Adult, Senior
Genders Eligible for Study:	Male
Accepts Healthy Volunteers:	No
Sampling Method:	Non-Probability Sample

Study Population

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

Criteria

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.

Contacts and Locations

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01391637

Locations


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

Sponsors and Collaborators

StemCells, Inc.

Investigators


Study Director:	Stephen Huhn, MD	StemCells, Inc.

More Information


Responsible Party:	StemCells, Inc.
ClinicalTrials.gov Identifier:	NCT01391637 History of Changes
Other Study ID Numbers:	CL-N02-PMD
Study First Received:	July 6, 2011
Last Updated:	May 11, 2016
Health Authority:	United States: Food and Drug Administration

Keywords provided by StemCells, Inc.:


Long term follow-up HuCNS-SC cells human central nervous system stem cells

Additional relevant MeSH terms:


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

ClinicalTrials.gov processed this record on September 23, 2016