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

January 13, 2015 updated by: StemCells, Inc.

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

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.

Study Overview

Status

Withdrawn

Conditions

Intervention / Treatment

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

Interventional

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Oregon
      • Portland, Oregon, United States, 97239
        • Oregon Health and Science University

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 6 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Other: HuCNS-SC
Biological: HuCNS-SC
Surgery to implant human CNS stem cells
Other Names:
  • stem cells
  • human neural stem cells
  • neural stem cells

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

StemCells, Inc.

Investigators

  • Principal Investigator: Nathan Selden, MD, PhD, Oregon Health and Science University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

November 1, 2010

Primary Completion (Actual)

April 1, 2011

Study Completion (Actual)

April 1, 2011

Study Registration Dates

First Submitted

November 5, 2010

First Submitted That Met QC Criteria

November 8, 2010

First Posted (Estimate)

November 10, 2010

Study Record Updates

Last Update Posted (Estimate)

January 15, 2015

Last Update Submitted That Met QC Criteria

January 13, 2015

Last Verified

January 1, 2015

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CL-N03-NCL

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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