Long-Term Follow-Up Study of Human Stem Cells Transplante... | NCT01391637

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

NCT01391637

Sponsor

StemCells, Inc.

Status

Completed

Last Update Posted

May 13, 2016Estimated

Enrollment

4Actual

Phase

Not provided

Conditions

Pelizaeus-Merzbacher Disease
PMD

Interventions

HuCNS-SC transplant in the lead-in phase

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

May 2016

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

Jun 2011

Primary Completion Date

Mar 2016Actual

Completion Date

Mar 2016Actual

First Submitted Date

Jul 6, 2011

First Submission Date that Met QC Criteria

Jul 8, 2011

First Posted Date

Jul 12, 2011Estimated

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

May 11, 2016

Last Update Posted Date

May 13, 2016Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

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 long term safety and preliminary effect of HuCNS-SC cells transplanted in subjects with Connatal Pelizaeus-Merzbacher Disease (PMD).

Detailed 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.

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

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 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

Name	Type	Description	Arm Group Labels	Other Names
HuCNS-SC transplant in the lead-in phase	Biological	Long-term safety follow-up study	HuCNS-SC transplanted subjects in the lead-in phase

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Incidence of serious adverse events (SAEs), results of physical and neurological examination, laboratory tests and vital signs.		4 years

Secondary Outcomes

Measure	Description	Time Frame
Preliminary efficacy using Bayley-III and Callier-Azusa Scale.	Changes compared to baseline	4 years
Changes in brain magnetic resonance imaging (MRI), electroencephalogram (EEG), seizure frequency and somato-sensory evoked potentials (SSEP).

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

Male

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

Not provided

Standard Ages

ChildAdultOlder Adult

Study Population

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

Sampling Method

Non-Probability Sample

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
UCSF Medical Center		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