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

StemCells, Inc.1 site in 1 countryNovember 2010

ConditionsNeuronal Ceroid Lipofuscinosis

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: Neuronal Ceroid Lipofuscinosis
Sponsor: StemCells, Inc.
Locations: 1
Primary Endpoint: number of participants with adverse events.
Status: Withdrawn
Last Updated: 11 years ago

Overview Investigators Eligibility Criteria Outcomes Sites Similar Trials

Overview

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.

Registry

clinicaltrials.gov

Start Date

November 2010

End Date

April 2011

Last Updated

11 years ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

StemCells, Inc.

Responsible Party

Sponsor

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