Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)

January 13, 2015 updated by: StemCells, Inc.

A Phase I Study of the Safety and Preliminary Effectiveness of Human CNS Stem Cells (HuCNS-SC) in Patients With Neuronal Ceroid Lipofuscinosis Caused by Palmitoyl Protein Thioesterase 1 (PPT1) or Tripeptidyl Peptidase 1 (TPP-I) Deficiency

Patients with infantile or late infantile NCL have either a reduced amount of, or are missing, the palmitoyl protein thioesterase 1 (PPT1) enzyme or the tripeptidyl peptidase 1 (TPP-I) enzyme. Human central nervous system stem cells (HuCNS-SC) are an investigational product derived from human brain cells. HuCNS-SC have been shown to survive and migrate within the brains of mice. When grown in the laboratory, HuCNS-SC have been shown to produce the PPT1 and TPP-I enzymes. In mice missing the PPT1 enzyme, HuCNS-SC have been shown to increase the amount of this enzyme in the brain, to reduce the amount of abnormal storage material in the brain, and to prevent the death of some neurons (a type of cell) in the brain.

Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress the immune system, and a series of follow-up assessments. The length of time from the start of screening through to the last follow-up visit will be approximately 13 months, with frequent visits to the study center during this time. After completion of this study, patients will be monitored for an additional 4 years under a separate long term follow-up protocol.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

6

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

1 year to 12 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

Patients MAY be eligible to participate in this research study if they:

  • Are age 18 months to 12 years old
  • Have a clinical diagnosis of infantile neuronal ceroid lipofuscinosis (INCL) or late infantile neuronal ceroid lipofuscinosis (LINCL)
  • Have a mutation of the CLN1 or CLN2 gene
  • Have severe cognitive, communication, behavior and language impairment

Exclusion Criteria:

Patients may not be eligible to participate in this research study if they:

  • Have cognitive, communication, behavior and language function less than that of a 1 year old
  • Have previously received an organ, tissue or bone marrow transplantation
  • Have previously participated in any gene or cell therapy study
  • Have infection with hepatitis virus, Cytomegalovirus, Epstein Barr Virus, or Human Immunodeficiency Virus (HIV)
  • Have a current or prior cancer
  • Have a bleeding disorder
  • Are 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: Non-Randomized
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: HuCNS-SC
human central nervous system stem cells
Procedure: Surgery to implant human CNS stem cells (HuCNS-SC)
single dose
Other Names:
  • HuCNS-SC
Drug: Medication to suppress the immune system
Immunosuppression for 12 months post transplant

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Safety
Time Frame: one year post transpant
one year post transpant

Secondary Outcome Measures

Outcome Measure
Time Frame
Preliminary efficacy
Time Frame: one year post transplant
one year post transplant

Collaborators and Investigators

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

Sponsor

StemCells, Inc.

Investigators

  • Principal Investigator: Robert Steiner, MD, 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.

General Publications

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

May 1, 2006

Primary Completion (Actual)

February 1, 2009

Study Completion (Actual)

September 1, 2009

Study Registration Dates

First Submitted

June 13, 2006

First Submitted That Met QC Criteria

June 13, 2006

First Posted (Estimate)

June 16, 2006

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-N001-05

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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