Safety Study of HLA-haplo Matched Allogenic Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis
Condition: Amyotrophic Lateral Sclerosis; Motor Neuron Disease; Neuromuscular Disease; Neurodegenerative Disease; Central Nervous System Disease
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Diseases; Central Nervous System Diseases; Sclerosis
Study type: Interventional
Phase: Phase 1

Summary: 
      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")
    

Detailed description: 
      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.
    

Intervention name(s): HYNR-CS-Allo
Intervention type(s): Genetic
Intervention description(s): 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.
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Amyotrophic Lateral Sclerosis; ALS; Neurodegenerative disease; Motor Neuron Disease; Central Nervous System Disease; Neuromuscular Disease; Bone marrow derived stem cell; stem cell therapy; intrathecal injection