Amyotrophic Lateral Sclerosis Clinical Trial
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 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 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.
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