Skip to main content
OpenTrials
Completed

NCT Number: NCT00337636

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

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.

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Neuronal Ceroid Lipofuscinosis Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurodegenerative Diseases Neuronal Ceroid-Lipofuscinoses Nutritional and Metabolic Diseases

Age range

18 month–12 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Oregon Health and Science University

Portland, Oregon, 97239, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

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

Procedure

single dose

Other names: HuCNS-SC

Medication to suppress the immune system

Drug

Immunosuppression for 12 months post transplant

Primary outcomes

  1. Safety

    Time frame: one year post transpant

Secondary outcomes

  1. Preliminary efficacy

    Time frame: one year post transplant

Sponsors and collaborators

Lead sponsor

StemCells, Inc.

Industry

Registry information

Official study 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

Important dates

Study start
2006
Primary completion
2009
Study completion
2009
First posted
Jun 16, 2006
Registry last updated
Jan 15, 2015

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.