Study of Human Central Nervous System (CNS) Stem Cells Tr... | NCT01005004

Studies
Study of Human Central Nervous System (CNS) Stem Cells Transplantation in Pelizaeus-Merzbacher Disease (PMD) Subjects

NCT01005004

Sponsor

StemCells, Inc.

Status

Completed

Last Update Posted

Jan 15, 2015Estimated

Enrollment

4Actual

Phase

Phase 1

Conditions

Pelizaeus-Merzbacher Disease

Interventions

HuCNS-SC cells

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Jan 2015

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Nov 2009

Primary Completion Date

Oct 2012Actual

Completion Date

Dec 2012Actual

First Submitted Date

Oct 28, 2009

First Submission Date that Met QC Criteria

Oct 28, 2009

First Posted Date

Oct 30, 2009Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jan 13, 2015

Last Update Posted Date

Jan 15, 2015Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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).

Detailed Description

Enrolled subjects will be transplanted with HuCNS-SC cells into the brain and will receive immunosuppression for nine months. The study observation period is for one year after transplant surgery. Thereafter, subjects will be enrolled in a long-term observational follow-up study for four years.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

4Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
HuCNS-SC cells	Experimental	Intracerebral implantation of HuCNS-SC via direct injection during surgery	Biological: HuCNS-SC cells

Interventions

Name	Type	Description	Arm Group Labels	Other Names
HuCNS-SC cells	Biological	intracerebral transplantation	HuCNS-SC cells

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Safety assessment through clinical neurological and MRI evaluation.		one year post transplant

Secondary Outcomes

Measure	Description	Time Frame
MRI examination for post-transplant myelination		one year post transplant

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Months

Maximum Age

5 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Stephen Huhn, MD	StemCells, Inc.	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of California, San Francisco		San Francisco	California	94143	United States

Condition Browse Module

MeSH Terms

ID	Term
D020371	Pelizaeus-Merzbacher Disease

Ancestor Terms

ID	Term
D020279	Hereditary Central Nervous System Demyelinating Diseases
D020739	Brain Diseases, Metabolic, Inborn
D001928	Brain Diseases, Metabolic
D001927	Brain Diseases

Browse Leaves

Not provided

Browse Branches

Not provided