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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
StemCells, Inc.
ClinicalTrials.gov Identifier:
NCT01391637
First received: July 6, 2011
Last updated: November 7, 2013
Last verified: November 2013

July 6, 2011
November 7, 2013
June 2011
February 2016   (final data collection date for primary outcome measure)
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 ]
Same as current
Complete list of historical versions of study NCT01391637 on ClinicalTrials.gov Archive Site
  • 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
Same as current
Not Provided
Not Provided
 
Long-Term Follow-Up Study of Human Stem Cells Transplanted in Subjects With Connatal Pelizaeus-Merzbacher Disease (PMD)
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).

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.

Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Not Provided
Non-Probability Sample

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

  • Pelizaeus-Merzbacher Disease
  • PMD
Biological: HuCNS-SC transplant in the lead-in phase
Long-term safety follow-up study
HuCNS-SC transplanted subjects in the lead-in phase
Subjects who had HuCNS-SC transplant in the lead-in phase study CL-N01-PMD
Intervention: Biological: HuCNS-SC transplant in the lead-in phase
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Active, not recruiting
4
June 2016
February 2016   (final data collection date for primary outcome measure)

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.
Male
Not Provided
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT01391637
CL-N02-PMD
No
StemCells, Inc.
StemCells, Inc.
Not Provided
Study Director: Stephen Huhn, MD StemCells, Inc.
StemCells, Inc.
November 2013

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