Safety Study of HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis
The purpose of this study is to evaluate the safety of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNR-CS-Allo inj"), through intrathecal delivery for the treatment in patients with amyotrophic lateral sclerosis(ALS).
This study is an open label, dose up and down study using the 3+3 design to assess the safety of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNR-CS-Allo inj")
|Amyotrophic Lateral Sclerosis Motor Neuron Disease Neuromuscular Disease Neurodegenerative Disease Central Nervous System Disease||Genetic: HYNR-CS-Allo||Phase 1|
|Study Design:||Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
|Official Title:||An Open-label, Phase 1 Trial for Safety Study of HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell("HYNR-CS-Allo Inj") Treatment in Amyotrophic Lateral Sclerosis(ALS)|
- The Incidence of any treatment related serious adverse events(SAE) [ Time Frame: 6 months ]
- ALS-Functional rating scales(ALS-FRS) [ Time Frame: 6 months ]
- Incidence & Degree of Adverse Events(AE) [ Time Frame: 6 months ]
|Study Start Date:||December 2012|
|Estimated Study Completion Date:||December 2017|
|Estimated Primary Completion Date:||October 2017 (Final data collection date for primary outcome measure)|
HYNR-CS-Allo inj. 2 times by intrathecal administration with 28 days interval.
The patients enrolled in the trial will be successively allocated into three cohorts for HYNR-CS-Allo inj., 0.25 X 10^6 cells/kg, 0.5 X 10^6 cells/kg, 1 X 10^6 cells/kg, according to the 3+3, up and down protocol design. The first treatment cohort will be 0.5 X 10^6 cells/kg dose cohort.
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease characterized by motor neuron loss. Despite of many trials for disease-modifying, no treatment has so far changed natural course of disease.
The investigators had performed the pre-clinical and clinical studies using autologous bone marrow-derived stem cells in ALS. In the investigators' results of clinical trial, intrathecal injection of autologous bone marrow-derived stem cells is safe and could slow down disease progression and might be used as a disease modifying strategy in patients with ALS.
In the new field, like cell therapy, it is an important issue whether a bone marrow derived mesenchymal stem cells can be used as an allograft. Many investigators had showed that the immunoprivileged and immunosuppressive properties of mesenchymal stem cells result from the absence of major histocompatibility class II antigens and the secretion of T helper type 2 cytokines.
One potential advantage of allogenic bone marrow derived cells could be avoiding the need for procedural delay before treatment. And it is also hypothesized that the function of autologous bone marrow derived cells could be impaired in patients with co-morbidities or advanced age.
This study is to evaluate safety of HYNR-CS-Allo inj(HLA-haplo matched Allogenic bone marrow-derived stem cells) in patients with ALS.
The patients enrolled in the trial will be successively allocated into three cohorts for HYNR-CS-Allo inj., 0.25 X 10^6 cells/kg, 0.5 X 10^6 cells/kg, 1 X 10^6 cells/kg, according to the 3+3, up and down protocol design. The first treatment cohort will be 0.5 X 106 cells/kg dose cohort. Only a maximum of six patients will be given a particular dosage.
The scheduled assessments and visits will be carried out over three periods: run-in period, treatment period, and follow-up period.
The run-in period includes the screening visit where a written informed consent is obtained and the screening period where patients are assessed for eligibility. It will be completed within 30 days prior to enrollment. The patients meeting inclusion criteria will start the treatment period.
During the treatment period, subjects will be administered HYNR-CS-Allo inj. 2 times by intrathecal administration with 28 days interval.
The Follow-up period starts once subjects complete the treatment period and will continue until the final follow-up visit.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01758510
|Korea, Republic of|
|Hanyang University Seoul Hospital, Cell Therapy Center for Neurologic Disorders|
|Seoul, Haengdang-dong, Seongdong-gu, Korea, Republic of, 133-792|
|Principal Investigator:||Seung Hyun Kim, M.D., Ph.D||Hanyang University Seoul Hospital|