Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...

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

This study has been completed.
Information provided by (Responsible Party):
StemCells, Inc. Identifier:
First received: June 13, 2006
Last updated: January 13, 2015
Last verified: January 2015

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

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: 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

Resource links provided by NLM:

Further study details as provided by StemCells, Inc.:

Primary Outcome Measures:
  • Safety [ Time Frame: one year post transpant ]

Secondary Outcome Measures:
  • Preliminary efficacy [ Time Frame: one year post transplant ]

Enrollment: 6
Study Start Date: May 2006
Study Completion Date: September 2009
Primary Completion Date: February 2009 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: HuCNS-SC
human central nervous system stem cells
Procedure: Surgery to implant human CNS stem cells (HuCNS-SC)
single dose
Other Name: HuCNS-SC
Drug: Medication to suppress the immune system
Immunosuppression for 12 months post transplant


Ages Eligible for Study:   18 Months to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00337636

United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
StemCells, Inc.
Principal Investigator: Robert Steiner, MD Oregon Health and Science University
  More Information

Additional Information:
Publications automatically indexed to this study by Identifier (NCT Number):
Responsible Party: StemCells, Inc. Identifier: NCT00337636     History of Changes
Other Study ID Numbers: CL-N001-05
Study First Received: June 13, 2006
Last Updated: January 13, 2015

Keywords provided by StemCells, Inc.:
Batten disease
Infantile Neuronal Ceroid Lipofuscinosis (INCL)
Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

Additional relevant MeSH terms:
Neuronal Ceroid-Lipofuscinoses
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lipid Metabolism Disorders
Metabolic Diseases processed this record on May 23, 2017