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

NCT ID: NCT01238315

Last Updated: 2015-01-15

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

WITHDRAWN

Clinical Phase

PHASE1

Study Classification

INTERVENTIONAL

Study Start Date

2010-11-30

Study Completion Date

2011-04-30

Brief Summary

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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

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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.

Conditions

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Neuronal Ceroid Lipofuscinosis

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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HuCNS-SC

Group Type OTHER

HuCNS-SC

Intervention Type BIOLOGICAL

Surgery to implant human CNS stem cells

Interventions

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HuCNS-SC

Surgery to implant human CNS stem cells

Intervention Type BIOLOGICAL

Other Intervention Names

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human neural stem cells stem cells neural stem cells

Eligibility Criteria

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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
Minimum Eligible Age

6 Months

Maximum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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StemCells, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Nathan Selden, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Oregon Health and Science University

Locations

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Oregon Health and Science University

Portland, Oregon, United States

Site Status

Countries

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United States

Related Links

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Other Identifiers

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CL-N03-NCL

Identifier Type: -

Identifier Source: org_study_id

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