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MEsenchymal StEm cells for Multiple Sclerosis (MESEMS): a randomized, double blind, cross-over phase I/II clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis
BACKGROUND: Multiple sclerosis (MS) is an inflammatory disease of the central nervous system with a degenerative component, leading to irreversible disability. Mesenchymal stem cells (MSC) have been shown to prevent inflammation and neurodegeneration in animal models of MS, but no large phase II cli...
Autores principales: | , , , , , , , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2019
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6507027/ https://www.ncbi.nlm.nih.gov/pubmed/31072380 http://dx.doi.org/10.1186/s13063-019-3346-z |
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author | Uccelli, Antonio Laroni, Alice Brundin, Lou Clanet, Michel Fernandez, Oscar Nabavi, Seyed Massood Muraro, Paolo A. Oliveri, Roberto S. Radue, Ernst W. Sellner, Johann Soelberg Sorensen, Per Sormani, Maria Pia Wuerfel, Jens Thomas Battaglia, Mario A. Freedman, Mark S. |
author_facet | Uccelli, Antonio Laroni, Alice Brundin, Lou Clanet, Michel Fernandez, Oscar Nabavi, Seyed Massood Muraro, Paolo A. Oliveri, Roberto S. Radue, Ernst W. Sellner, Johann Soelberg Sorensen, Per Sormani, Maria Pia Wuerfel, Jens Thomas Battaglia, Mario A. Freedman, Mark S. |
author_sort | Uccelli, Antonio |
collection | PubMed |
description | BACKGROUND: Multiple sclerosis (MS) is an inflammatory disease of the central nervous system with a degenerative component, leading to irreversible disability. Mesenchymal stem cells (MSC) have been shown to prevent inflammation and neurodegeneration in animal models of MS, but no large phase II clinical trials have yet assessed the exploratory efficacy of MSC for MS. METHODS/DESIGN: This is an academic, investigator-initiated, randomized, double-blind, placebo-compared phase I/II clinical trial with autologous, bone-marrow derived MSC in MS. Enrolled subjects will receive autologous MSC at either baseline or at week 24, through a cross-over design. Primary co-objectives are to test safety and efficacy of MSC treatment compared to placebo at 6 months. Secondary objectives will evaluate the efficacy of MSC at clinical and MRI levels. In order to overcome funding constraints, the MEsenchymal StEm cells for Multiple Sclerosis (MESEMS) study has been designed to merge partially independent clinical trials, following harmonized protocols and sharing some key centralized procedures, including data collection and analyses. DISCUSSION: Results will provide patients and the scientific community with data on the safety and efficacy of MSC for MS. The innovative approach utilized to obtain funds to support the MESEMS trial could represent a new model to circumvent limitation of funds encountered by academic trials. TRIAL REGISTRATION: Andalusia: NCT01745783, registered on Dec 10, 2012. Badalona: NCT02035514 EudraCT, 2010–024081–21. Registered on 2012. Canada: ClinicalTrials.gov, NCT02239393. Registered on September 12, 2014. Copenhagen: EudraCT, 2012–000518-13. Registered on June 21, 2012. Italy: EudraCT, 2011–001295-19, and ClinicalTrials.gov, NCT01854957. Retrospectively registered on May 16, 2013. London: Eudra CT 2012–002357-35, and ClinicalTrials.gov, NCT01606215. Registered on May 25, 2012. Salzburg: EudraCT, 2015–000137-78. Registered on September 15, 2015. Stockholm: ClinicalTrials.gov, NCT01730547. Registered on November 21, 2012. Toulouse: ClinicalTrials.gov, NCT02403947. Registered on March 31, 2015. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1186/s13063-019-3346-z) contains supplementary material, which is available to authorized users. |
format | Online Article Text |
id | pubmed-6507027 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2019 |
publisher | BioMed Central |
record_format | MEDLINE/PubMed |
spelling | pubmed-65070272019-05-13 MEsenchymal StEm cells for Multiple Sclerosis (MESEMS): a randomized, double blind, cross-over phase I/II clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis Uccelli, Antonio Laroni, Alice Brundin, Lou Clanet, Michel Fernandez, Oscar Nabavi, Seyed Massood Muraro, Paolo A. Oliveri, Roberto S. Radue, Ernst W. Sellner, Johann Soelberg Sorensen, Per Sormani, Maria Pia Wuerfel, Jens Thomas Battaglia, Mario A. Freedman, Mark S. Trials Study Protocol BACKGROUND: Multiple sclerosis (MS) is an inflammatory disease of the central nervous system with a degenerative component, leading to irreversible disability. Mesenchymal stem cells (MSC) have been shown to prevent inflammation and neurodegeneration in animal models of MS, but no large phase II clinical trials have yet assessed the exploratory efficacy of MSC for MS. METHODS/DESIGN: This is an academic, investigator-initiated, randomized, double-blind, placebo-compared phase I/II clinical trial with autologous, bone-marrow derived MSC in MS. Enrolled subjects will receive autologous MSC at either baseline or at week 24, through a cross-over design. Primary co-objectives are to test safety and efficacy of MSC treatment compared to placebo at 6 months. Secondary objectives will evaluate the efficacy of MSC at clinical and MRI levels. In order to overcome funding constraints, the MEsenchymal StEm cells for Multiple Sclerosis (MESEMS) study has been designed to merge partially independent clinical trials, following harmonized protocols and sharing some key centralized procedures, including data collection and analyses. DISCUSSION: Results will provide patients and the scientific community with data on the safety and efficacy of MSC for MS. The innovative approach utilized to obtain funds to support the MESEMS trial could represent a new model to circumvent limitation of funds encountered by academic trials. TRIAL REGISTRATION: Andalusia: NCT01745783, registered on Dec 10, 2012. Badalona: NCT02035514 EudraCT, 2010–024081–21. Registered on 2012. Canada: ClinicalTrials.gov, NCT02239393. Registered on September 12, 2014. Copenhagen: EudraCT, 2012–000518-13. Registered on June 21, 2012. Italy: EudraCT, 2011–001295-19, and ClinicalTrials.gov, NCT01854957. Retrospectively registered on May 16, 2013. London: Eudra CT 2012–002357-35, and ClinicalTrials.gov, NCT01606215. Registered on May 25, 2012. Salzburg: EudraCT, 2015–000137-78. Registered on September 15, 2015. Stockholm: ClinicalTrials.gov, NCT01730547. Registered on November 21, 2012. Toulouse: ClinicalTrials.gov, NCT02403947. Registered on March 31, 2015. ELECTRONIC SUPPLEMENTARY MATERIAL: The online version of this article (10.1186/s13063-019-3346-z) contains supplementary material, which is available to authorized users. BioMed Central 2019-05-09 /pmc/articles/PMC6507027/ /pubmed/31072380 http://dx.doi.org/10.1186/s13063-019-3346-z Text en © The Author(s). 2019 Open AccessThis article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made. The Creative Commons Public Domain Dedication waiver (http://creativecommons.org/publicdomain/zero/1.0/) applies to the data made available in this article, unless otherwise stated. |
spellingShingle | Study Protocol Uccelli, Antonio Laroni, Alice Brundin, Lou Clanet, Michel Fernandez, Oscar Nabavi, Seyed Massood Muraro, Paolo A. Oliveri, Roberto S. Radue, Ernst W. Sellner, Johann Soelberg Sorensen, Per Sormani, Maria Pia Wuerfel, Jens Thomas Battaglia, Mario A. Freedman, Mark S. MEsenchymal StEm cells for Multiple Sclerosis (MESEMS): a randomized, double blind, cross-over phase I/II clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis |
title | MEsenchymal StEm cells for Multiple Sclerosis (MESEMS): a randomized, double blind, cross-over phase I/II clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis |
title_full | MEsenchymal StEm cells for Multiple Sclerosis (MESEMS): a randomized, double blind, cross-over phase I/II clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis |
title_fullStr | MEsenchymal StEm cells for Multiple Sclerosis (MESEMS): a randomized, double blind, cross-over phase I/II clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis |
title_full_unstemmed | MEsenchymal StEm cells for Multiple Sclerosis (MESEMS): a randomized, double blind, cross-over phase I/II clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis |
title_short | MEsenchymal StEm cells for Multiple Sclerosis (MESEMS): a randomized, double blind, cross-over phase I/II clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis |
title_sort | mesenchymal stem cells for multiple sclerosis (mesems): a randomized, double blind, cross-over phase i/ii clinical trial with autologous mesenchymal stem cells for the therapy of multiple sclerosis |
topic | Study Protocol |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6507027/ https://www.ncbi.nlm.nih.gov/pubmed/31072380 http://dx.doi.org/10.1186/s13063-019-3346-z |
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