Mesenchymal Stem Cells in Multiple Sclerosis (MSCIMS)

Phase 1

Completed

• Conditions: Multiple Sclerosis
• Interventions: Procedure: MSC Treatment

• Registration Number: NCT00395200
• Lead Sponsor: University of Cambridge

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

Study Timeline

• Study First Submitted: 2006-11-01
• Study First Submitted QC: 2006-11-01
• Study First Posted: 2006-11-02
• Study Start: 2008-07
• Primary Completion: 2010-12
• Study Completion: 2010-12
• Status Verified: 2011-10
• Last Update Submitted: 2011-10-22
• Last Update Posted: 2011-10-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
MSC Treatment	MSC Treatment	-

Primary Outcome Measures

Name	Time	Method
Adverse events	0,1,2,3,4,12 and 52 weeks post treatment

Secondary Outcome Measures

Name	Time	Method
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

Trial Locations

Locations (2)

University College London Institute of Neurology; 🇬🇧 London, United Kingdom

University of Cambridge Dept of Clinical Neurosciences; 🇬🇧 Cambridge, Cambridgeshire, United Kingdom