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

This study has been withdrawn prior to enrollment.

(Lack of timely patient accrual)

Sponsor:

Information provided by (Responsible Party):

StemCells, Inc.

ClinicalTrials.gov Identifier:

NCT01238315

First received: November 5, 2010

Last updated: May 24, 2012

Last verified: May 2012

History of Changes

Tracking Information

First Received Date ^ICMJE

November 5, 2010

Last Updated Date

May 24, 2012

Start Date ^ICMJE

November 2010

Primary Completion Date

April 2011 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

number of participants with adverse events. [ Time Frame: one year following transplantation ] [ Designated as safety issue: Yes ]

Safety will be measured by number of adverse events and changes in neurological status.

Original Primary Outcome Measures ^ICMJE

Safety assessment through clinical, neurological and MRI evaluation. [ Time Frame: one year following transplantation ] [ Designated as safety issue: Yes ]

Change History

Complete list of historical versions of study NCT01238315 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS [ Time Frame: one year following transplantation ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^ICMJE

Improvement in neurological, neuropsychological development, MRI and MRS [ Time Frame: one year following transplantation ] [ Designated as safety issue: No ]

Current Other Outcome Measures ^ICMJE

Not Provided

Original Other Outcome Measures ^ICMJE

Not Provided

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Nervous System Stem Cell Transplantation in Subjects With Non-Refractory Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis

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.

Study Type ^ICMJE

Interventional

Study Phase

Phase 1

Study Design ^ICMJE

Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Condition ^ICMJE

Neuronal Ceroid Lipofuscinosis

Intervention ^ICMJE

Biological: HuCNS-SC

Surgery to implant human CNS stem cells

Other Names:

human neural stem cells
stem cells
neural stem cells

Study Arm (s)

HuCNS-SC

Intervention: Biological: HuCNS-SC

Publications *

Not Provided

* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Withdrawn

Enrollment ^ICMJE

Completion Date

April 2011

Primary Completion Date

April 2011 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

6 Months to 6 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States

Administrative Information

NCT Number ^ICMJE

NCT01238315

Other Study ID Numbers ^ICMJE

CL-N03-NCL

Has Data Monitoring Committee

Yes

Responsible Party

StemCells, Inc.

Study Sponsor ^ICMJE

StemCells, Inc.

Collaborators ^ICMJE

Not Provided

Investigators ^ICMJE

Principal Investigator:

Nathan Selden, MD, PhD

Oregon Health and Science University

Information Provided By

StemCells, Inc.

Verification Date

May 2012

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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