Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
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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. |
ClinicalTrials.gov Identifier: NCT01238315 |
Recruitment Status :
Withdrawn
(Lack of timely patient accrual)
First Posted : November 10, 2010
Last Update Posted : January 15, 2015
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Neuronal Ceroid Lipofuscinosis | Biological: HuCNS-SC | Phase 1 |
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:
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- 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.
- Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS [ Time Frame: one year following transplantation ]

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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

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
United States, Oregon | |
Oregon Health and Science University | |
Portland, Oregon, United States, 97239 |
Principal Investigator: | Nathan Selden, MD, PhD | Oregon Health and Science University |
Responsible Party: | StemCells, Inc. |
ClinicalTrials.gov 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 |
Batten's disease INCL LINCL Neuronal Ceroid Lipofuscinosis NCL |
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 |