Safety/Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in ALS
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|ClinicalTrials.gov Identifier: NCT03214146|
Recruitment Status : Active, not recruiting
First Posted : July 11, 2017
Last Update Posted : May 16, 2019
The purpose of this study is to evaluate the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj"), through intrathecal delivery for the repeated treatment after 6 months of first treatment in patients with amyotrophic lateral sclerosis(ALS).
This study is an open label, single-dose study to assess the safety and efficacy of HLA-haplo matched Allogenic Bone Marrow Derived stem cells("HYNRCS-Allo-ALS-02 inj")
|Condition or disease||Intervention/treatment||Phase|
|Amyotrophic Lateral Sclerosis||Biological: HYNRCS-Allo inj||Phase 1|
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 and efficacy of repeated treatment after 6 months of first HYNRCS-Allo-ALS-02 inj(HLA-haplo matched Allogenic bone marrow-derived stem cells) treatment in patients with ALS.
The patients enrolled in the trial will be successively allocated into single cohort for HYNRCS-Allo-ALS-02 inj., 1.0 X 10^6 cells/kg, according to the protocol design. 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 56 days prior to enrollment. The patients meeting inclusion criteria will start the treatment period.
During the treatment period, subjects will be administered HYNRCS-Allo-ALS-02 inj. 2 times(first treatment) by intrathecal administration with 28 days interval and there will be repeated treatment after 6 months of the first treatment.
The Follow-up period starts once subjects complete the treatment period and will continue until the final follow-up visit.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||6 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open-label, Phase 1 Trial for Safety and Efficacy Study of 2nd Cycle Treatment After 6 Months of 1st Cycle HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell("HYNR-CS-Allo Inj") Treatment in Amyotrophic Lateral Sclerosis(ALS)|
|Actual Study Start Date :||February 1, 2017|
|Actual Primary Completion Date :||March 25, 2019|
|Estimated Study Completion Date :||October 2021|
Experimental: HYNRCS-Allo inj.
2 cycles of HYNRCS-Allo inj. with 6 months interval through intrathecal injection.
*1 cycle of HYNRCS-Allo inj. is 2 times administration with 28 days interval by intrathecal.
Biological: HYNRCS-Allo inj
The patients enrolled in the trial will be successively allocated into single cohort for HYNRCS-Allo inj., 1.0 X 10^6 cells/kg according to the protocol design.
Other Name: HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell
- The Incidence of any treatment related serious adverse events(SAE) [ Time Frame: 12 months ]
- ALS-Functional rating scales(ALS-FRS) [ Time Frame: 12 months ]
- Incidence & Degree of Adverse Events(AE) [ Time Frame: 12 months ]
- PRA test to identify generation status of HLA antibody [ Time Frame: 12 months ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03214146
|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|