Status:
COMPLETED
Study of HuCNS-SC Cells in Patients With Infantile or Late Infantile Neuronal Ceroid Lipofuscinosis (NCL)
Lead Sponsor:
StemCells, Inc.
Conditions:
Neuronal Ceroid Lipofuscinosis
Eligibility:
All Genders
18-12 years
Phase:
PHASE1
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 c...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
May 1 2006
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2009
Estimated Enrollment :
6 Patients enrolled
Trial Details
Trial ID
NCT00337636
Start Date
May 1 2006
End Date
September 1 2009
Last Update
January 15 2015
Active Locations (1)
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1
Oregon Health and Science University
Portland, Oregon, United States, 97239