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

Completed · Phase 1

Conditions studied: Neuronal Ceroid Lipofuscinosis

In brief

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.

Key facts

Study ID

NCT00337636

Run by

StemCells, Inc.

People needed

6

Starts

2006-05-01

Expected to finish

2009-09-01

Last updated by the study team

2015-01-15

Who can join

Age: 2 and older, up to 12. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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

You may not qualify if…

• 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

Where it is running

• Oregon Health and Science University — Portland, Oregon, United States

Full record on ClinicalTrials.gov

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