Safety and Efficacy of Intravenous Autologous Mesenchymal Stem Cells for MS: a Phase 2 Proof of Concept Study
Condition: Multiple Sclerosis
MeSH terms: Multiple Sclerosis; Sclerosis
Study type: Interventional
Phase: Phase 2

Summary: 
      The mechanism of action of MSC relies on their ability to modulate pathogenic immune
      responses and provide neuroprotection through the release of anti-apoptotic, anti-oxidant and
      trophic factors as demonstrated by in-vitro and in-vivo preclinical studies.

      Patients will be randomized to receive immediate vs. delayed treatment with either a dose
      equal to 1-2 millions/kg of body weight of autologous MSC, or equivalent volume of suspension
      media at baseline. At week 24 treatments will be reversed.

      The primary outcome of this study is to evaluate:

        -  Treatment's safety within one year from MSC administration by measuring the number,
           time-frame and severity of adverse events and

        -  Treatment's activity in terms of reduction in total number of gadolinium-enhancing
           lesions (GEL) by magnetic resonance imaging (MRI) scans.

      Secondary outcomes are to gain preliminary information on the efficacy of the experimental
      treatment in terms of combined MRI activity and clinical efficacy (incidence of relapses and
      disability progression).
    

Detailed description: 
      The mechanism of action of Mesenchymal Stem Cells (MSCs) relies on their ability to modulate
      pathogenic immune responses and provide neuroprotection through the release of
      anti-apoptotic, anti-oxidant and trophic factors as demonstrated by in vitro and in vivo
      preclinical studies.

      Patients will be randomized to receive immediate vs. delayed treatment with either a dose
      equal to 1-2 millions/kg of body weight of autologous MSC, or equivalent volume of suspension
      media at baseline. At 6 months treatments will be reversed.

      The primary outcome of this study is to evaluate

        -  treatment's safety within one year from MSC administration by measuring the the number,
           time-frame and severity of adverse event and

        -  treatment's activity in terms of reduction in the total number of contrast-gadolinium
           enhancing lesions (GEL) by magnetic resonance imaging (MRI) scans.

      Secondary outcomes are to gain preliminary information on the efficacy of the experimental
      treatment in terms of combined MRI activity and clinical efficacy (incidence of relapses and
      disability progression).
    

Intervention name(s): Mesenchymal Stem Cells
Intervention type(s): Biological
Intervention description(s): Mesenchymal Stem Cells in Plasma-Lyte A (Baxter) suspension media, containing 5% Human Albumin and 10% dimethylsulfoxide (DMSO, total volume of 5mL DMSO in final cell product) and autologous MSCs at a dose of 1 to 2 x 106 MSC/Kg participant's body weight at randomization.
Matching placebo Plasma-Lyte A (Baxter) suspension media, containing 5% Human Albumin and 10% DMSO (total volume of 5mL DMSO in final cell product).
Allocation: Randomized
Intervention model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary purpose: Treatment
Keywords: Multiple Sclerosis; Mesenchymal Stem Cells; MSC