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

Sponsor

StemCells, Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT00337636

Collaborator

(none)

Enrollment

Location

Arm

Duration (Months)

0.1

Patients Per Site Per Month

Study Details

Study Description

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 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 or Disease	Intervention/Treatment	Phase
Neuronal Ceroid Lipofuscinosis	Procedure: Surgery to implant human CNS stem cells (HuCNS-SC) Drug: Medication to suppress the immune system	Phase 1

Study Design

Study Type:

Interventional

Actual Enrollment :

6 participants

Allocation:

Non-Randomized

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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

Study Start Date :

May 1, 2006

Actual Primary Completion Date :

Feb 1, 2009

Actual Study Completion Date :

Sep 1, 2009

Arms and Interventions

Arm	Intervention/Treatment
Experimental: HuCNS-SC human central nervous system stem cells	Procedure: Surgery to implant human CNS stem cells (HuCNS-SC) single dose Other Names: HuCNS-SC Drug: Medication to suppress the immune system Immunosuppression for 12 months post transplant

Arm

Intervention/Treatment

Experimental: HuCNS-SC

human central nervous system stem cells

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

single dose

Other Names:

HuCNS-SC

Drug: Medication to suppress the immune system

Immunosuppression for 12 months post transplant

Outcome Measures

Primary Outcome Measures

Safety [one year post transpant]

Secondary Outcome Measures

Preliminary efficacy [one year post transplant]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Months to 12 Years

Sexes Eligible for Study:

All

Accepts 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