NCT01005004

Brief Summary

The purpose of this study is to determine the safety and preliminary effectiveness of human central nervous system stem cells (HuCNS-SC®) transplantation in patients 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 phase_1

Timeline
Completed

Started Nov 2009

Typical duration for phase_1

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

First Submitted

Initial submission to the registry

October 28, 2009

Completed
2 days until next milestone

First Posted

Study publicly available on registry

October 30, 2009

Completed
2 days until next milestone

Study Start

First participant enrolled

November 1, 2009

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2012

Completed
2 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2012

Completed
Last Updated

January 15, 2015

Status Verified

January 1, 2015

Enrollment Period

2.9 years

First QC Date

October 28, 2009

Last Update Submit

January 13, 2015

Conditions

Pelizaeus-Merzbacher Disease

Keywords

PMDstem cellsPelizaeus-Merzbacher Diseasehuman stem cellscentral nervous system stem cells

Outcome Measures

Primary Outcomes (1)

  • Safety assessment through clinical neurological and MRI evaluation.

    one year post transplant

Secondary Outcomes (1)

  • MRI examination for post-transplant myelination

    one year post transplant

Study Arms (1)

HuCNS-SC cells

EXPERIMENTAL

Intracerebral implantation of HuCNS-SC via direct injection during surgery

Biological: HuCNS-SC cells

Interventions

HuCNS-SC cellsBIOLOGICAL

intracerebral transplantation

Also known as: Human central nervous system stem cells
HuCNS-SC cells

Eligibility Criteria

Age6 Months - 5 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Confirmed clinical diagnosis of connatal PMD
  • Molecular genetic confirmation of mutation in the proteolipid protein 1 (PLP1) gene
  • MRI consistent with PMD as interpreted by a qualified neuroradiologist

You may not qualify if:

  • Other significant congenital brain abnormality not related to PMD
  • Previous participation in gene transfer or cell transplant trial
  • Presence of neurological signs and symptoms not consistent with PMD
  • Current or prior malignancy
  • Prior organ, tissue or bone marrow transplant

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

University of California, San Francisco

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
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 28, 2009

First Posted

October 30, 2009

Study Start

November 1, 2009

Primary Completion

October 1, 2012

Study Completion

December 1, 2012

Last Updated

January 15, 2015

Record last verified: 2015-01

Locations

US(1)