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

Tracking Information

First Received Date ^ICMJE

June 13, 2006

Last Updated Date

October 27, 2009

Start Date ^ICMJE

May 2006

Primary Completion Date

February 2009 (final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Safety [ Time Frame: one year post transpant ] [ Designated as safety issue: Yes ]

Original Primary Outcome Measures ^ICMJE

Same as current

Change History

Complete list of historical versions of study NCT00337636 on ClinicalTrials.gov Archive Site

Current Secondary Outcome Measures ^ICMJE

Preliminary efficacy [ Time Frame: one year post transplant ] [ Designated as safety issue: No ]

Original Secondary Outcome Measures ^ICMJE

Same as current

Descriptive Information

Brief Title ^ICMJE

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

Official Title ^ICMJE

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

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.

Detailed Description

Study Phase

Phase I

Study Type ^ICMJE

Interventional

Study Design ^ICMJE

Treatment, Non-Randomized, Open Label, Single Group Assignment, Safety/Efficacy Study

Condition ^ICMJE

Neuronal Ceroid Lipofuscinosis

Intervention ^ICMJE

Procedure: Surgery to implant human CNS stem cells (HuCNS-SC)
Drug: Medication to suppress the immune system

Study Arms / Comparison Groups

Publications *

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status ^ICMJE

Completed

Enrollment ^ICMJE

Completion Date

September 2009

Primary Completion Date

February 2009 (final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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

Gender

Both

Ages

18 Months to 12 Years

Accepts Healthy Volunteers

Contacts ^ICMJE

Contact information is only displayed when the study is recruiting subjects

Location Countries ^ICMJE

United States

Administrative Information

NCT ID ^ICMJE

NCT00337636

Responsible Party

Stephen Huhn, MD, StemCells, Inc.

Study ID Numbers ^ICMJE

CL-N001-05

Study Sponsor ^ICMJE

StemCells, Inc.

Collaborators ^ICMJE

Investigators ^ICMJE

Principal Investigator:

Robert Steiner, MD

Oregon Health and Science University

Information Provided By

StemCells, Inc.

Verification Date

October 2009

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP