Mesenchymal Stem Cells in Multiple Sclerosis (MSCIMS)
Condition: Multiple Sclerosis
MeSH terms: Multiple Sclerosis; Sclerosis
Study type: Interventional
Phase: Phase 1/Phase 2

Summary: 
      Hypothesis: Intravenous administration of bone marrow-derived autologous adult human
      mesenchymal stem cells is a safe novel therapeutic approach for patients with multiple
      sclerosis.

      Mesenchymal Stem Cells in Multiple Sclerosis (MSCIMS) is a phase I/IIA trial designed to
      establish the safety of intravenous administration of bone marrow-derived autologous adult
      human mesenchymal stem cells to patients with multiple sclerosis.
    

Detailed description: 
      Disease under investigation: Multiple Sclerosis

      Phase: I/IIA

      Number of patients: 10

      Design: 18 month cross over, single treatment at 6 months

      Intervention: Administration of bone marrow-derived autologous mesenchymal stem cells

      Route of administration: Intravenous

      Dose: Up to 2,000,000 Mesenchymal Stem Cells per kilogram

      Source of patients: Referrals accepted from Neurologists in East Anglia and North London, UK

      Referral Criteria: (all 3 required)

        1. Clinically definite multiple sclerosis

        2. Expanded Kurtzke Disability Status Score 2.0 - 6.5 (inclusive)

        3. Evidence of optic nerve damage by

             -  history of optic neuritis, or

             -  relative afferent pupillary defect, or

             -  optic atrophy on fundoscopy, or

             -  abnormal visual evoked potential from either or both eyes suggestive of
                demyelination

      Primary Objective: Establish the safety of intravenously administered bone marrow-derived
      autologous mesenchymal stem cells at a dose of up to 2,000,000 cells/kg over 12 months by
      monitoring adverse reactions.

      Secondary Objectives: Explore the efficacy of intravenously administered bone marrow-derived
      autologous mesenchymal stem cells at a dose of up to 2,000,000 cells/kg over 12 months on
      visual function by clinical, neurophysiological, and imaging assessments.

      Outcome Measures:

        1. Primary

             -  Adverse events

        2. Secondary

             -  Visual function (acuity and colour)

             -  Visual evoked potential latency

             -  Optic nerve Magnetisation Transfer Ratio

             -  Retinal nerve fibre layer thickness (by optical coherence tomography)

             -  Brain lesion Magnetisation Transfer Ratio

             -  MRI brain T1 hypointensity load

             -  T cell response suppression

        3. Tertiary

             -  Multiple Sclerosis Functional Composite Score

             -  Expanded Kurtzke Disability Status Score
    

Intervention name(s): MSC Treatment
Intervention type(s): Procedure
Intervention description(s): Intravenous administration of up to 2x10^6 autologous MSCs per kg
Other intervention name(s): Mesenchymal Stem Cells; Multipotent Mesenchymal Stem Cells; Multipotent Mesenchymal Stromal Cells
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Multiple Sclerosis; Safety; Therapeutics; Mesenchymal Stem Cells; Multipotent Mesenchymal Stromal Cells; Optic Neuritis