Stem Cell Therapy for Patients With Multiple Sclerosis Failing Alternate Approved Therapy- A Randomized Study
Condition: Multiple Sclerosis
MeSH terms: Multiple Sclerosis; Sclerosis
Study type: Interventional
Phase: Phase 2

Summary: 
      Multiple sclerosis (MS) is at onset an immune-mediated demyelinating disease. In most cases,
      it starts as a relapsing-remitting disease with distinct attacks and no symptoms between
      flares. Over years or decades, virtually all cases transition into a progressive disease in
      which insidious and slow neurologic deterioration occurs with or without acute flares.
      Relapsing-remitting disease is often responsive to immune suppressive or modulating
      therapies, while immune based therapies are generally ineffective in patients with a
      progressive clinical course. This clinical course and response to immune suppression, as well
      as neuropathology and neuroimaging studies, suggest that disease progression is associated
      with axonal atrophy. Disability correlates better with measures of axonal atrophy than immune
      mediated demyelination. Therefore, immune based therapies, in order to be effective, need to
      be started early in the disease course while MS is predominately an immune-mediated and
      inflammatory disease. While current immune based therapies delay disability, no intervention
      has been proven to prevent progressive disability. We propose, as a randomized study,
      autologous unmanipulated PBSCT using a conditioning regimen of cyclophosphamide and rabbit
      antithymocyte globulin (rATG) versus FDA approved standard of care (i.e. interferon,
      glatiramer acetate, mitoxantrone, natalizumab, fingolimod, or tecfidera) in patients with
      inflammatory (relapsing) MS despite treatment with alternate approved therapy.
    

Detailed description: 
      To assess the efficacy of autologous PBSCT versus FDA approved standard of care ( i.e.
      interferon, glatiramer acetate, mitoxantrone, natalizumab, fingolimod, or tecfidera) for
      inflammatory multiple sclerosis (MS) failing failing alternate approved therapy. The
      endpoints to be considered in this study are:

      2.1 Primary Endpoint:

      Disease progression, defined as a 1 point increase in the Expanded Disability Status Scale
      (EDSS) on consecutive evaluations at least 6 months apart and not due to a non-MS disease
      process. Patients will be followed for 5 years after randomization.
    

Intervention name(s): Hematopoietic Stem Cell Therapy; Standard treatment with a conventional drug
Intervention type(s): Procedure; Drug
Intervention description(s): After mobilization and harvest of stem cells, stem cells will be infused following conditioning regimen; Standard treatment with a conventional drug is the treatment with one of the following drugs: Avonex (interferon beta 1a), Betaseron (interferon beta 1b), Copaxone (glatiramer acetate), Aubagio (teriflunomide), Tysabri (natalizumab), or Gilenya (fingolimod)
Other intervention name(s): stem cell infusion; standard of care
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: stem cell, autoimmune disease, multiple sclerosis