Neuronal Ceroid Lipofuscinosis Clinical Trial
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
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.
n/a
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
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