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Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis

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ClinicalTrials.gov Identifier: NCT01238315
Recruitment Status : Withdrawn (Lack of timely patient accrual)
First Posted : November 10, 2010
Last Update Posted : January 15, 2015
Sponsor:
Information provided by (Responsible Party):
StemCells, Inc.

Study Description
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information
Brief Summary:
The purpose of this Phase Ib study is to determine if "Human Central Nervous System Stem Cells"(HuCNS-SC) is safe to be transplanted in subjects with infantile and late infantile neuronal ceroid lipofuscinosis. The study will also measure post-transplantation disease progression.

Condition or disease Intervention/treatment Phase
Neuronal Ceroid Lipofuscinosis Biological: HuCNS-SC Phase 1

Detailed Description:
Participation in this study will involve screening assessments, surgery to implant HuCNS-SC, medication to suppress immune system and a series of follow-up assessments.

Study Design
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase Ib Study of the Safety and Preliminary Efficacy of Allogeneic Intracerebral Human Central Nervous System Stem Cell Transplantation in Subjects With Non-Refractory Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis
Study Start Date : November 2010
Actual Primary Completion Date : April 2011
Actual Study Completion Date : April 2011

Resource links provided by the National Library of Medicine

U.S. FDA Resources

Arms and Interventions
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Arm Intervention/treatment
HuCNS-SC Biological: HuCNS-SC
Surgery to implant human CNS stem cells
Other Names:
  • human neural stem cells
  • stem cells
  • neural stem cells


Outcome Measures
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Primary Outcome Measures :
  1. number of participants with adverse events. [ Time Frame: one year following transplantation ]
    Safety will be measured by number of adverse events and changes in neurological status.


Secondary Outcome Measures :
  1. Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS [ Time Frame: one year following transplantation ]

Eligibility Criteria
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

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Ages Eligible for Study:   6 Months to 6 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Age 6 months to 6 years
  • Male or female
  • Clinical diagnosis of Infantile neuronal ceroid lipofuscinosis or late infantile neuronal ceroid lipofuscinosis
  • CLN1 or CLN2 mutation

Exclusion Criteria:

  • Previously received an organ, tissue or bone marrow transplantation
  • Previously participated in any gene or cell therapy study
  • Infection with hepatitis virus, cytomegalovirus, Epstein-Barr virus, or Human Immunodeficiency Virus (HIV)
  • Current or prior cancer
  • Bleeding disorder
  • Unable to have an MRI scan
Contacts and Locations
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01238315


Locations
United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
StemCells, Inc.
Investigators
Principal Investigator: Nathan Selden, MD, PhD Oregon Health and Science University
More Information
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Study Description Study Design Arms and Interventions Outcome Measures Eligibility Criteria Contacts and Locations More Information

Additional Information:
Related Info  This link exits the ClinicalTrials.gov site
Infantile and Late Infantile Neuronal Ceroid Lipofuscinosis  This link exits the ClinicalTrials.gov site

Responsible Party: StemCells, Inc.
ClinicalTrials.gov Identifier: NCT01238315     History of Changes
Other Study ID Numbers: CL-N03-NCL
First Posted: November 10, 2010    Key Record Dates
Last Update Posted: January 15, 2015
Last Verified: January 2015

Keywords provided by StemCells, Inc.:
Batten's disease
INCL
LINCL
Neuronal Ceroid Lipofuscinosis
NCL

Additional relevant MeSH terms:
Neuronal Ceroid-Lipofuscinoses
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Genetic Diseases, Inborn
 Lipidoses
Lipid Metabolism, Inborn Errors
Metabolism, Inborn Errors
Lipid Metabolism Disorders
Metabolic Diseases