Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
This study has been withdrawn prior to enrollment.
(Lack of timely patient accrual)
Sponsor:
StemCells, Inc.
Information provided by (Responsible Party):
StemCells, Inc.
ClinicalTrials.gov Identifier:
NCT01238315
First received: November 5, 2010
Last updated: January 13, 2015
Last verified: January 2015
- Full Text View
- Tabular View
- No Study Results Posted
- Disclaimer
- How to Read a Study Record
Purpose
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 | Intervention | Phase |
|---|---|---|
|
Neuronal Ceroid Lipofuscinosis |
Biological: HuCNS-SC |
Phase 1 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
Chanarin-Dorfman syndrome
Kufs disease
cholesteryl ester storage disease
congenital neuronal ceroid lipofuscinosis
infantile neuronal ceroid lipofuscinosis
juvenile Batten disease
late-infantile neuronal ceroid lipofuscinosis
Genetic and Rare Diseases Information Center resources:
Adult Neuronal Ceroid Lipofuscinosis
Ceroid Storage Disease
Neuronal Ceroid Lipofuscinosis
U.S. FDA Resources
Further study details as provided by StemCells, Inc.:
Primary Outcome Measures:
- number of participants with adverse events. [ Time Frame: one year following transplantation ] [ Designated as safety issue: Yes ]Safety will be measured by number of adverse events and changes in neurological status.
Secondary Outcome Measures:
- Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS [ Time Frame: one year following transplantation ] [ Designated as safety issue: No ]
| Enrollment: | 0 |
| Study Start Date: | November 2010 |
| Study Completion Date: | April 2011 |
| Primary Completion Date: | April 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| HuCNS-SC |
Biological: HuCNS-SC
Surgery to implant human CNS stem cells
Other Names:
|
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.
Eligibility| Ages Eligible for Study: | 6 Months to 6 Years (Child) |
| Genders Eligible for Study: | Both |
| 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
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, see Learn About Clinical Studies.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01238315
Please refer to this study by its ClinicalTrials.gov identifier: 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
Additional Information:
| Responsible Party: | StemCells, Inc. |
| ClinicalTrials.gov Identifier: | NCT01238315 History of Changes |
| Other Study ID Numbers: | CL-N03-NCL |
| Study First Received: | November 5, 2010 |
| Last Updated: | January 13, 2015 |
| Health Authority: | United States: Food and Drug Administration |
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 |
ClinicalTrials.gov processed this record on September 28, 2016