NCT00337636

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
6

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started May 2006

Typical duration for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

May 1, 2006

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

June 13, 2006

Completed
3 days until next milestone

First Posted

Study publicly available on registry

June 16, 2006

Completed
2.6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2009

Completed
7 months until next milestone

Study Completion

Last participant's last visit for all outcomes

September 1, 2009

Completed
Last Updated

January 15, 2015

Status Verified

January 1, 2015

Enrollment Period

2.8 years

First QC Date

June 13, 2006

Last Update Submit

January 13, 2015

Conditions

Neuronal Ceroid Lipofuscinosis

Keywords

NCLINCLLINCLBatten diseaseInfantile Neuronal Ceroid Lipofuscinosis (INCL)Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

Outcome Measures

Primary Outcomes (1)

  • Safety

    one year post transpant

Secondary Outcomes (1)

  • Preliminary efficacy

    one year post transplant

Study Arms (1)

HuCNS-SC

EXPERIMENTAL

human central nervous system stem cells

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

Interventions

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

single dose

Also known as: HuCNS-SC
HuCNS-SC
Medication to suppress the immune systemDRUG

Immunosuppression for 12 months post transplant

HuCNS-SC

Eligibility Criteria

Age18 Months - 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

Related Publications (1)

  • Selden NR, Al-Uzri A, Huhn SL, Koch TK, Sikora DM, Nguyen-Driver MD, Guillaume DJ, Koh JL, Gultekin SH, Anderson JC, Vogel H, Sutcliffe TL, Jacobs Y, Steiner RD. Central nervous system stem cell transplantation for children with neuronal ceroid lipofuscinosis. J Neurosurg Pediatr. 2013 Jun;11(6):643-52. doi: 10.3171/2013.3.PEDS12397. Epub 2013 Apr 12.

    PMID: 23581634DERIVED

Related Links

MeSH Terms

Conditions

Neuronal Ceroid-Lipofuscinoses

Condition Hierarchy (Ancestors)

Heredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesNervous System DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesLipidosesLipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Robert Steiner, MD

    Oregon Health and Science University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 13, 2006

First Posted

June 16, 2006

Study Start

May 1, 2006

Primary Completion

February 1, 2009

Study Completion

September 1, 2009

Last Updated

January 15, 2015

Record last verified: 2015-01

Locations

US(1)