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Sponsored by: |
StemCells, Inc. |
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Information provided by: | StemCells, Inc. |
ClinicalTrials.gov Identifier: | NCT00337636 |
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 |
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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, Dose Comparison, 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 |
Ages Eligible for Study: | 18 Months to 12 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Patients MAY be eligible to participate in this research study if they:
Exclusion Criteria:
Patients may not be eligible to participate in this research study if they:
United States, Oregon | |
Oregon Health and Science University | |
Portland, Oregon, United States, 97239 |
Principal Investigator: | Robert Steiner, MD | Oregon Health and Science University |
Study ID Numbers: | CL-N001-05 |
Study First Received: | June 13, 2006 |
Last Updated: | January 25, 2008 |
ClinicalTrials.gov Identifier: | NCT00337636 |
Health Authority: | United States: Food and Drug Administration |
NCL INCL LINCL |
Batten disease Infantile Neuronal Ceroid Lipofuscinosis (INCL) Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL) |
Lipid Metabolism, Inborn Errors Ceroid lipofuscinosis, neuronal Metabolic Diseases Neurodegenerative Diseases Metabolism, Inborn Errors Heredodegenerative Disorders, Nervous System Genetic Diseases, Inborn |
Neuronal Ceroid-Lipofuscinoses Spielmeyer-Vogt disease Ceroid lipofuscinosis, neuronal 3, Juvenile Lipidoses Metabolic disorder Lipid Metabolism Disorders |
Nervous System Diseases |