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

Sponsor:	StemCells, Inc.
Information provided by:	StemCells, Inc.
ClinicalTrials.gov Identifier:	NCT00337636

Purpose

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	Intervention	Phase
Neuronal Ceroid Lipofuscinosis	Procedure: Surgery to implant human CNS stem cells (HuCNS-SC) Drug: Medication to suppress the immune system	Phase I

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study
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

Resource links provided by NLM:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis juvenile Batten disease primary carnitine deficiency

MedlinePlus related topics: Surgery

U.S. FDA Resources

Further study details as provided by StemCells, Inc.:

Primary Outcome Measures:

Safety [ Time Frame: one year post transpant ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Preliminary efficacy [ Time Frame: one year post transplant ] [ Designated as safety issue: No ]

Enrollment:	6
Study Start Date:	May 2006
Study Completion Date:	September 2009
Primary Completion Date:	February 2009 (Final data collection date for primary outcome measure)

Intervention Details:

single dose

Immunosuppression for 12 months post transplant

Eligibility

Ages Eligible for Study:	18 Months to 12 Years
Genders Eligible for Study:	Both
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

Please refer to this study by its ClinicalTrials.gov identifier: NCT00337636

Locations

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

Sponsors and Collaborators

StemCells, Inc.

Investigators

Principal Investigator:

Robert Steiner, MD

Oregon Health and Science University

More Information

Additional Information:

Click here for information about StemCells, Inc. This link exits the ClinicalTrials.gov site

No publications provided

Responsible Party:	StemCells, Inc. ( Stephen Huhn, MD )
Study ID Numbers:	CL-N001-05
Study First Received:	June 13, 2006
Last Updated:	October 27, 2009
ClinicalTrials.gov Identifier:	NCT00337636 History of Changes
Health Authority:	United States: Food and Drug Administration

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:

Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Metabolic Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn

Nervous System Diseases
Neuronal Ceroid-Lipofuscinoses
Lipidoses
Neurodegenerative Diseases
Lipid Metabolism Disorders

ClinicalTrials.gov processed this record on November 05, 2009