Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis
Phase 1
Withdrawn
- Conditions
- Neuronal Ceroid Lipofuscinosis
- Interventions
- Biological: HuCNS-SC
- Registration Number
- NCT01238315
- Lead Sponsor
- 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.
- 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.
Recruitment & Eligibility
- Status
- WITHDRAWN
- Sex
- All
- Target Recruitment
- Not specified
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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description HuCNS-SC HuCNS-SC -
- Primary Outcome Measures
Name Time Method number of participants with adverse events. one year following transplantation Safety will be measured by number of adverse events and changes in neurological status.
- Secondary Outcome Measures
Name Time Method Number of subjects with improvement in neurological, neuropsychological development, MRI and MRS one year following transplantation
Trial Locations
- Locations (1)
Oregon Health and Science University
🇺🇸Portland, Oregon, United States