MedTrace Logo

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

NCT00337636 · Status: COMPLETED · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 6

Last updated 2015-01-15

No results posted yet for this study

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.

Conditions

  • Neuronal Ceroid Lipofuscinosis

Interventions

PROCEDURE

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

single dose

DRUG

Medication to suppress the immune system

Immunosuppression for 12 months post transplant

Sponsors & Collaborators

  • StemCells, Inc.

    lead INDUSTRY

Principal Investigators

  • Robert Steiner, MD · Oregon Health and Science University

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Months
Max Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2006-05-31
Primary Completion
2009-02-28
Completion
2009-09-30

Countries

  • United States

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00337636 on ClinicalTrials.gov