EMMES: Autologous Mesenchymal Stromal Cells for Multiple Sclerosis

Sponsor

Banc de Sang i Teixits (Other)

Overall Status

Completed

CT.gov ID

NCT02495766

Collaborator

Vall d'Hebron Research Institute (VHIR) (Other)

Enrollment

Location

Arms

42.2

Actual Duration (Months)

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This study evaluates the effect of cryopreserved autologous adult bone-marrow mesenchymal stromal cells (BM-MSC) in patients with active multiple sclerosis, compared to placebo.

Patients will be allocated to one of the 2 treatment arms (BM-MSC or placebo)and at month 6, the treatment will be crossed to receive the other product. The objective is to assess the safety of a single infusion BM-MSC, and to explore its efficacy in these patients.

Patients will be evaluated at month 12 and will be followed-up for a total of 3 years.

Condition or Disease	Intervention/Treatment	Phase
Relapsing-Remitting Multiple Sclerosis Secondary Progressive Multiple Sclerosis	Drug: XCEL-MC-ALPHA Drug: Placebo	Phase 1/Phase 2

Detailed Description

To date, there is no effective therapy to cure multiple sclerosis (MS). Immunomodulatory therapies are useful in reducing the frequency of inflammatory processes (relapses) but don't delay significantly the progression of the disease, prevent long term disability or induce the repair of damaged tissue. This proposal contemplates the use of adult autologous bone marrow mesenchymal stromal cells (BM-MSC) as an alternative therapeutic strategy to treat patients with active MS. This is a randomized, double blind, crossover clinical trial in which 8 patients with active forms of MS and moderate disability will enter the trial with the primary objective of assessing the safety and tolerability of a single intravenous infusion of BM-MSC. Secondary objectives are to assess the efficacy by gadolinium enhancing lesions though magnetic resonance imaging, neurophysiological effects and immunological effects. Once randomized, patients will undergo BM extraction and once confirmed the availability of the needed dose, they will be randomized to one of the 2 treatment arms (BM-MSC named XCEL-MC-ALPHA, or placebo). XCEL-MC-ALPHA will be cryopreserved for all patients regardless the allocated arm. At month 6, the treatment will be crossed. Patients will be evaluated at month 12 and will be followed-up for a total of 3 years.

Study Design

Study Type:

Interventional

Actual Enrollment :

8 participants

Allocation:

Randomized

Intervention Model:

Crossover Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Primary Purpose:

Treatment

Official Title:

Treatment of Autologous Mesenchymal Stem Cells Derived From Bone Marrow as a Potential Therapeutic Strategy for the Treatment of Multiple Sclerosis

Actual Study Start Date :

May 11, 2015

Actual Primary Completion Date :

Nov 27, 2017

Actual Study Completion Date :

Nov 15, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Treatment A: XCEL-MC-ALPHA/Placebo Single infusion of cryopreserved bone-marrow adult mesenchymal stromal cells followed by placebo infusion at month 6.	Drug: XCEL-MC-ALPHA Single infusion Other Names: Bone-marrow mesenchymal stromal cells Drug: Placebo Single infusion
Experimental: Treatment B: Placebo/XCEL-MC-ALPHA Single infusion of placebo followed by cryopreserved bone-marrow adult mesenchymal stromal cells infusion at month 6.	Drug: XCEL-MC-ALPHA Single infusion Other Names: Bone-marrow mesenchymal stromal cells Drug: Placebo Single infusion

Arm

Intervention/Treatment

Experimental: Treatment A: XCEL-MC-ALPHA/Placebo

Single infusion of cryopreserved bone-marrow adult mesenchymal stromal cells followed by placebo infusion at month 6.

Drug: XCEL-MC-ALPHA

Single infusion

Other Names:

Bone-marrow mesenchymal stromal cells

Drug: Placebo

Single infusion

Experimental: Treatment B: Placebo/XCEL-MC-ALPHA

Single infusion of placebo followed by cryopreserved bone-marrow adult mesenchymal stromal cells infusion at month 6.

Drug: XCEL-MC-ALPHA

Single infusion

Other Names:

Bone-marrow mesenchymal stromal cells

Drug: Placebo

Single infusion

Outcome Measures

Primary Outcome Measures

Adverse events [12 months]
Safety profile

Secondary Outcome Measures

Cumulative number of MRI Gd-enhancing lesions [12 months]
Imaging procedure
Multiple Sclerosis Outbreaks [12 months]
Medical assessment
Expanded Disability Status Scale (EDDS) score [12 months]
Quantification of disability
Cumulative number of lesions visualized on T2 sequence [12 months]
Imaging procedure

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 60 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Patients between 18 and 60 years of age
Patients with MS
Relapsing-remitting or secondary progressive MS
Patients to whom are not indicated or are not in a position to initiate treatment with disease-modifying drugs
Expanded Disability Status Scale (EDDS) score <6.5
Nine T2 lesions at least
Active multiple sclerosis as defined either by 1 outbreak in the last year or at least one Gadolinium-enhancing lesion in the last 6 months
Signed informed consent form