Safety and Efficacy Study of HuCNS-SC in Subjects With Ne... | NCT01238315

Studies
Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis

NCT01238315

Sponsor

StemCells, Inc.

Status

Withdrawn

Last Update Posted

Jan 15, 2015Estimated

Enrollment

0Actual

Phase

Phase 1

Conditions

Neuronal Ceroid Lipofuscinosis

Interventions

HuCNS-SC

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

Withdrawn

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Lack of timely patient accrual

Expanded Access Info

Start Date

Nov 2010

Primary Completion Date

Apr 2011Actual

Completion Date

Apr 2011Actual

First Submitted Date

Nov 5, 2010

First Submission Date that Met QC Criteria

Nov 8, 2010

First Posted Date

Nov 10, 2010Estimated

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 Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis. The study will also measure post-transplantation disease progression.

Detailed Description

Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress immune system and a series of follow-up assessments.

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

0Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
HuCNS-SC	Other		Biological: HuCNS-SC

Interventions

Name	Type	Description	Arm Group Labels	Other Names
HuCNS-SC	Biological	Surgery to implant human CNS stem cells	HuCNS-SC

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
number of participants with adverse events.	Safety will be measured by number of adverse events and changes in neurological status.	one year following transplantation

Secondary Outcomes

Measure	Description	Time Frame
Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS		one year following transplantation

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

Age 6 months to 6 years
Male or female
Clinical diagnosis of Infantile neuronal ceroid lipofuscinosis or late infantile neuronal ceroid lipofuscinosis
CLN1 or CLN2 mutation

Exclusion Criteria:

Previously received an organ, tissue or bone marrow transplantation
Previously participated in any gene or cell therapy study
Infection with hepatitis virus, cytomegalovirus, Epstein-Barr virus, or Human Immunodeficiency Virus (HIV)
Current or prior cancer
Bleeding disorder
Unable to have an MRI scan

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Months

Maximum Age

6 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Nathan Selden, MD, PhD	Oregon Health and Science University	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Oregon Health and Science University		Portland	Oregon	97239	United States

Label	URL
Related Info	View source

Condition Browse Module

MeSH Terms

ID	Term
D009472	Neuronal Ceroid-Lipofuscinoses

Ancestor Terms

ID	Term
D020271	Heredodegenerative Disorders, Nervous System
D019636	Neurodegenerative Diseases
D009422	Nervous System Diseases
D030342	Genetic Diseases, Inborn

Browse Leaves

Not provided

Browse Branches

Not provided