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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00337636
Recruitment Status : Completed
First Posted : June 16, 2006
Last Update Posted : January 15, 2015
Sponsor:
Information provided by (Responsible Party):
StemCells, Inc.

Study Description
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Brief Summary:

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.


Condition or disease Intervention/treatment Phase
Neuronal Ceroid Lipofuscinosis Procedure: Surgery to implant human CNS stem cells (HuCNS-SC) Drug: Medication to suppress the immune system Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Study Start Date : May 2006
Actual Primary Completion Date : February 2009
Actual Study Completion Date : September 2009

Resource links provided by the National Library of Medicine


Arms and Interventions
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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 Name: HuCNS-SC

Drug: Medication to suppress the immune system
Immunosuppression for 12 months post transplant


Outcome Measures
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Primary Outcome Measures :
  1. Safety [ Time Frame: one year post transpant ]

Secondary Outcome Measures :
  1. Preliminary efficacy [ Time Frame: one year post transplant ]

Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Months to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00337636


Locations
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United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
StemCells, Inc.
Investigators
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Principal Investigator: Robert Steiner, MD Oregon Health and Science University
More Information
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Additional Information:

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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Responsible Party: StemCells, Inc.
ClinicalTrials.gov Identifier: NCT00337636    
Other Study ID Numbers: CL-N001-05
First Posted: June 16, 2006    Key Record Dates
Last Update Posted: January 15, 2015
Last Verified: January 2015
Keywords provided by StemCells, Inc.:
NCL
INCL
LINCL
 Batten disease
Infantile Neuronal Ceroid Lipofuscinosis (INCL)
Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)
Additional relevant MeSH terms:
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Neuronal Ceroid-Lipofuscinoses
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Genetic Diseases, Inborn
 Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lipid Metabolism Disorders
Metabolic Diseases