Autologous Adult Human Mesenchymal Stem Cells: a Neuroprotective Therapy for Multiple Sclerosis
Overview
- Phase
- Phase 1
- Intervention
- Not specified
- Conditions
- Multiple Sclerosis
- Sponsor
- University of Cambridge
- Enrollment
- 10
- Locations
- 2
- Primary Endpoint
- Adverse events
- Status
- Completed
- Last Updated
- 14 years ago
Overview
Brief 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
Investigators
Peter Connick
Research Associate
University of Cambridge
Eligibility Criteria
Inclusion Criteria
- •Clinically definite multiple sclerosis
- •Expanded Kurtzke Disability Status Score 2.0 - 6.5 (inclusive)
- •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
- •Prolonged visual evoked potential P100 latency with preserved waveform
- •T2 lesion on MRI optic nerve
- •Retinal nerve fibre layer thickness on optical coherence tomography \> 40 microns
Exclusion Criteria
- •Age \< 18 years
- •Age \> 65 years
- •Patient lacks capacity to give informed consent
- •Presence of a severe bleeding disorder
- •Planning a pregnancy during the trial period
- •Current MS disease modifying therapy
Outcomes
Primary Outcomes
Adverse events
Time Frame: 0,1,2,3,4,12 and 52 weeks post treatment
Secondary Outcomes
- Visual function (acuity and colour)(12 and 52 weeks post treatment)
- Visual evoked potential latency(12 and 52 weeks post treatment)
- Optic nerve Magnetisation Transfer Ratio(12 and 52 weeks post treatment)
- Retinal nerve fibre layer thickness (by optical coherence tomography)(12 and 52 weeks post treatment)
- Brain lesion Magnetisation Transfer Ratio(12 and 52 weeks post treatment)
- MRI brain T1 hypointensity load(12 and 52 weeks post treatment)
- Multiple Sclerosis Functional Composite Score(12 and 52 weeks post treatment)
- Expanded Kurtzke Disability Status Score(12 and 52 weeks post treatment)