Back to resultsSafety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
Primary Purpose
Neuronal Ceroid Lipofuscinosis
Status
Withdrawn
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
HuCNS-SC
Sponsored by
About this trial
This is an interventional treatment trial for Neuronal Ceroid Lipofuscinosis focused on measuring Batten's disease, INCL, LINCL, Neuronal Ceroid Lipofuscinosis, NCL
Eligibility 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
Sites / Locations
- Oregon Health and Science University
Arms of the Study
Arm 1
Arm Type
Other
Arm Label
HuCNS-SC
Arm Description
Outcomes
Primary Outcome Measures
number of participants with adverse events.
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
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01238315
Brief Title
Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
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 Type
Interventional
2. Study Status
Record Verification Date
January 2015
Overall Recruitment Status
Withdrawn
Why Stopped
Lack of timely patient accrual
Study Start Date
November 2010 (undefined)
Primary Completion Date
April 2011 (Actual)
Study Completion Date
April 2011 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
StemCells, Inc.
4. Oversight
Data Monitoring Committee
Yes
5. Study Description
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.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Neuronal Ceroid Lipofuscinosis
Keywords
Batten's disease, INCL, LINCL, Neuronal Ceroid Lipofuscinosis, NCL
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
HuCNS-SC
Arm Type
Other
Intervention Type
Biological
Intervention Name(s)
HuCNS-SC
Other Intervention Name(s)
human neural stem cells, stem cells, neural stem cells
Intervention Description
Surgery to implant human CNS stem cells
Primary Outcome Measure Information:
Title
number of participants with adverse events.
Description
Safety will be measured by number of adverse events and changes in neurological status.
Time Frame
one year following transplantation
Secondary Outcome Measure Information:
Title
Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS
Time Frame
one year following transplantation
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Nathan Selden, MD, PhD
Organizational Affiliation
Oregon Health and Science University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Oregon Health and Science University
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
12. IPD Sharing Statement
Learn more about this trial
Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
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