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

StemCells, Inc

Status and phase

Completed

Phase 1

Conditions

Neuronal Ceroid Lipofuscinosis

Treatments

Drug: Medication to suppress the immune system

Procedure: Surgery to implant human CNS stem cells (HuCNS-SC)

Study type

Interventional

Funder types

Industry

Identifiers

NCT00337636

CL-N001-05

Details and patient eligibility

About

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.

Enrollment

6 patients

Sex

All

Ages

18 months to 12 years old

Volunteers

No Healthy Volunteers

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