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

Pelizaeus-Merzbacher Disease Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01391637
• Other study ID #: CL-N02-PMD
• Status: Completed
• Phase: Phase 1
• First received: July 6, 2011
• Last updated: May 11, 2016
• Start date: June 2011
• Est. completion date: March 2016

Study information

• Verified date: May 2016
• Source: StemCells, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Observational

Clinical Trial Summary

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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 4
• Est. completion date: March 2016
• Est. primary completion date: March 2016
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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 Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Pelizaeus-Merzbacher Disease
• PMD

Intervention

Biological:
• HuCNS-SC transplant in the lead-in phase
Long-term safety follow-up study

Locations

Country	Name	City	State
United States	UCSF Medical Center	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
StemCells, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.		4 years	Yes
Secondary	Preliminary efficacy using Bayley-III and Callier-Azusa Scale.	Changes compared to baseline	4 years	No
Secondary	Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP).	Changes compared to baseline	4 years	No