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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT01238315 |
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Date of registration:
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05/11/2010 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
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Scientific title:
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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 |
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Date of first enrolment:
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November 2010 |
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Target sample size:
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0 |
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Recruitment status: |
Withdrawn |
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URL:
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http://clinicaltrials.gov/show/NCT01238315 |
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Study type:
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Interventional |
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Study design:
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Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Nathan Selden, MD, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Oregon Health and Science University |
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Key inclusion & exclusion criteria
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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
Age minimum:
6 Months
Age maximum:
6 Years
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Neuronal Ceroid Lipofuscinosis
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Intervention(s)
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Biological: HuCNS-SC
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Primary Outcome(s)
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number of participants with adverse events.
[Time Frame: one year following transplantation]
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Secondary Outcome(s)
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Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS
[Time Frame: one year following transplantation]
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Secondary ID(s)
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CL-N03-NCL
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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