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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

6 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-05-31

Study Completion Date

2009-09-30

Brief Summary

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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.

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Detailed Description

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Conditions

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Neuronal Ceroid Lipofuscinosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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HuCNS-SC

human central nervous system stem cells

Group Type EXPERIMENTAL

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

Intervention Type PROCEDURE

single dose

Medication to suppress the immune system

Intervention Type DRUG

Immunosuppression for 12 months post transplant

Interventions

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

single dose

Intervention Type PROCEDURE

Medication to suppress the immune system

Immunosuppression for 12 months post transplant

Intervention Type DRUG

Other Intervention Names

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HuCNS-SC

Eligibility Criteria

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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

Minimum Eligible Age

18 Months

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No