NCT01391637

Brief 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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
4

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jun 2011

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2011

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

July 6, 2011

Completed
6 days until next milestone

First Posted

Study publicly available on registry

July 12, 2011

Completed
4.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2016

Completed
Last Updated

May 13, 2016

Status Verified

May 1, 2016

Enrollment Period

4.8 years

First QC Date

July 6, 2011

Last Update Submit

May 11, 2016

Conditions

Pelizaeus-Merzbacher DiseasePMD

Keywords

Long term follow-upHuCNS-SC cellshuman central nervous system stem cells

Outcome Measures

Primary Outcomes (1)

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

    4 years

Secondary Outcomes (2)

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

    4 years

  • Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP).

    4 years

Study Arms (1)

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

Interventions

HuCNS-SC transplant in the lead-in phaseBIOLOGICAL

Long-term safety follow-up study

HuCNS-SC transplanted subjects in the lead-in phase

Eligibility Criteria

Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

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

You may qualify if:

  • Subjects who received HuCNS-SC cells under Protocol CL-N01-PMD

You may not qualify if:

  • Subjects who received off-protocol immunosuppressive medications.
  • Subjects who are concurrently enrolled in another investigational study.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

UCSF Medical Center

San Francisco, California, 94143, United States

Location

MeSH Terms

Conditions

Pelizaeus-Merzbacher Disease

Condition Hierarchy (Ancestors)

Hereditary Central Nervous System Demyelinating DiseasesBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesLeukoencephalopathiesDemyelinating DiseasesGenetic Diseases, X-LinkedGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolism, Inborn ErrorsMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Stephen Huhn, MD

    StemCells, Inc.

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 6, 2011

First Posted

July 12, 2011

Study Start

June 1, 2011

Primary Completion

March 1, 2016

Study Completion

March 1, 2016

Last Updated

May 13, 2016

Record last verified: 2016-05

Locations

US(1)