Safety and Efficacy Study of Autologous Bone Marrow Derived Stem Cell Treatment in Amyotrophic Lateral Sclerosis
Condition: Amyotrophic Lateral Sclerosis; ALS
MeSH terms: Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Sclerosis
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      The purpose of this study is to evaluate the safety and efficacy of autologous bone
      marrow-derived stem cells("HYNR-CS inj"), through intrathecal delivery for the treatment in
      patients with ALS.

      This study consists of 2 steps. First step is a safety study of the intrathecal(IT) injection
      of "HYNR-CS inj" in 8 patients with ALS. In this phase 1 study, AE, laboratory test, physical
      examination, vital signs, Electrocardiogram, and Chest X-Ray examination were evaluated in
      terms of safety.

      Second step is to compare the efficacy and safety between test group and control group of
      total 64 patients with ALS.
    

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.

      We have performed the pre-clinical and clinical studies using autologous bone marrow-derived
      stem cells in ALS. We could get the evidence that autologous bone marrow-derived stem cells
      have dose-dependent effects on SOD1 mice via intrathecal injection. In our results of
      clinical trial, intrathecal injection of autologous bone marrow-derived stem cells could slow
      down disease progression and might be used as a disease modifying strategy in patients with
      ALS.

      This study was designed as a single center, randomized, open-label, parallel-group, 2-stage
      study, and targeted at patients diagnosed with Amyotrophic Lateral Sclerosis(Lou Gehrig's
      disease). The study consisted of Stage-1 study for safety evaluation and Stage-2 study for
      efficacy and safety evaluation of the study drug, and at Stage 1, 7 subjects eligible for the
      inclusion/exclusion criteria received safety evaluation for 28 days of study drug
      administration in twice under the protocol, and then followed Stage 2. To decide whether the
      study can be proceeded in 2 stages, ADR(CTCAE Version 3.0, ≥grade 3) should not appear in
      initial 7 subjects.

      Data obtained from subjects of this study were analyzed into three: Safety Analysis,
      ITT(Intent-To-Treat) Analysis, and PP(Per Protocol) Analysis. However, in case of phase 1,
      only safety analysis was conducted, and in case of phase 2, all of safety, ITT, and PP
      analyses were conducted.

      For ITT Analysis, all the subjects whose data on primary efficacy endpoint could be obtained
      following the administration of investigational drug were analyzed in analysis among subjects
      who were administered the investigational drug once at least. Also, Modified ITT Analysis,
      including 7 subjects at Stage 1, was carried out.
    

Intervention name(s): HYNR-CS inj; Control group
Intervention type(s): Biological; Other
Intervention description(s): Intrathecal injection with 1ml/10kg of body weight at an interval of 26 days.; No treatment of HYNR-CS inj
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Amyotrophic lateral sclerosis; ALS; Motor neuron disease; MND; HYNR-CS inj; Autologous bone marrow derived stem cell; Intrathecal injection