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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01238315
Recruitment Status : Withdrawn (Lack of timely patient accrual)
First Posted : November 10, 2010
Last Update Posted : January 15, 2015
Information provided by (Responsible Party):
StemCells, Inc.

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 0 participants
Allocation: N/A
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

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

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 ]

Information from the National Library of Medicine

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, Learn About Clinical Studies.

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

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

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 identifier (NCT number): NCT01238315

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United States, Oregon
Oregon Health and Science University
Portland, Oregon, United States, 97239
Sponsors and Collaborators
StemCells, Inc.
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Principal Investigator: Nathan Selden, MD, PhD Oregon Health and Science University
Additional Information:
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Responsible Party: StemCells, Inc. Identifier: NCT01238315    
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
Neuronal Ceroid Lipofuscinosis
Additional relevant MeSH terms:
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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