Mesenchymal Stem Cell Transplantation in MS (CMM-EM)

Autologous Mesenchymal Stem Cell Transplantation in Multiple Sclerosis: a Randomized, Double-blind, Crossover With Placebo Phase II Study

The study is a randomized Phase II study, masked and crossed-over with placebo to evaluate the safety and tolerability of autologous mesenchymal stem cell transplantation in patients with active multiple sclerosis

Study Overview

• Status: Terminated
• Conditions: Multiple Sclerosis
• Intervention / Treatment: Biological: autologous mesenchymal stem cells

• Study Type: Interventional
• Enrollment (Actual): 9
• Phase: Phase 2

Contacts and Locations

Study Locations

• Spain
  • Barcelona, Spain, 08036
    • Neurology Service, Hospital Clinic de barcelona

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 50 years (ADULT)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Inflammatory forms of MS

   1. Relapsing-remitting MS (RRMS) patients
   2. Secondary progressive MS (SPMS) patients with continued relapses
   3. Primary progressive MS (PPMS) patients with enhancing MRI lesions and positive CSF (oligoclonal banding)
2. Age 18-50 years
3. Disease duration >= 2 and >= 10 years
4. EDSS 3.0 - 6.5

5. Progression, continued relapses or worsening MRI after at least a year of attempted therapy evidenced by:

   1. Increase of >= 1 EDSS point (if baseline EDSS <= 5.0) or 0.5 EDSS points (if baseline EDSS >= 5.5), or quantifiable, objective evidence of equivalent progression
   2. >= 1 moderate-severe relapses in past 18 months
   3. >= 1 Gadolinium enhancing lesions (double or triple dose Gadolinium)
   4. >= 1 new T2 lesion
   5. For PPMS only, >= 1 Gadolinium enhancing lesions
6. Has given informed consent to participate in the study.

Exclusion Criteria:

1. SPMS without ongoing relapses
2. PPMS without positive CSF or Gadolinium enhancing lesions
3. <= 3 months since treatment with any immunosuppressive therapy
4. <=1 month since last treatment with interferon-B or glatiramer acetate
5. Corticosteroid treatment <= 30 days
6. Relapse <= 60 days
7. History of cancer or clinical or laboratory results indicative of severe systemic diseases, including infection for HIV, Hepatitis B or C
8. Any metallic or electronic device that precludes from undergoing MRI
9. Pregnancy or lactation
10. Current treatment with an investigational therapy

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: RANDOMIZED
• Interventional Model: CROSSOVER
• Masking: DOUBLE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: autologous mesenchymal stem cell; A single infusion of up to 2 million cells per Kg of autologous mesenchymal stem cells vs suspension media. The treatment will be reversed at 6 months	Biological: autologous mesenchymal stem cells; A randomized double-blind, crossover study comparing treatment with autologous MSC vs. suspension media on patients with active MS

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate the safety as number of severe events along 1 year, and efficacy in terms of cumulative number of gadolinium-enhancing lesions in MRI at 6 months and at the end of the study	The coprimary endpoints were safety and efficacy in terms of cumulative number of gadolinium-enhancing lesions in MRI	12 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
To evaluate effects on MS disease activity measured by: clinical variables, MRI, OCT, immunological analysis and quality of life scales	clinical outcomes (number of relapses and change in the EDSS); MRI-based measures and OCT. Immunological evaluation as exploratory analysis	12 months

Collaborators and Investigators

• Sponsor: Albert Saiz
• Collaborators: Instituto de Salud Carlos III
• Investigators: Principal Investigator: Albert Saiz, MD, Hospital Clinic of Barcelona

Publications and helpful links

General Publications

• Llufriu S, Sepulveda M, Blanco Y, Marin P, Moreno B, Berenguer J, Gabilondo I, Martinez-Heras E, Sola-Valls N, Arnaiz JA, Andreu EJ, Fernandez B, Bullich S, Sanchez-Dalmau B, Graus F, Villoslada P, Saiz A. Randomized placebo-controlled phase II trial of autologous mesenchymal stem cells in multiple sclerosis. PLoS One. 2014 Dec 1;9(12):e113936. doi: 10.1371/journal.pone.0113936. eCollection 2014.

Study record dates

Study Major Dates

• Study Start: December 1, 2010
• Primary Completion (ACTUAL): June 1, 2013
• Study Completion (ACTUAL): December 1, 2013

Study Registration Dates

• First Submitted: October 25, 2010
• First Submitted That Met QC Criteria: October 25, 2010
• First Posted (ESTIMATE): October 26, 2010

Study Record Updates

• Last Update Posted (ESTIMATE): February 13, 2014
• Last Update Submitted That Met QC Criteria: February 12, 2014
• Last Verified: February 1, 2014

More Information

Terms related to this study

• Keywords: Multiple Sclerosis
• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Multiple Sclerosis; Sclerosis
• Other Study ID Numbers: CMM-EM