1. Efficacy and safety of autologous haematopoietic stem cell transplantation versus alemtuzumab, ocrelizumab, ofatumumab or cladribine in relapsing remitting multiple sclerosis (StarMS): protocol for a randomised controlled trial
- Author
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Gavin Giovannoni, Stephen J Walters, David Paling, Olga Ciccarelli, Cindy Cooper, Eli Silber, Martin Duddy, Hannah Hunter, David Hunt, Basil Sharrack, Paul Gallagher, Ian Galea, Richard Nicholas, Annalena Venneri, Neil Robertson, Carolyn Young, Jeremy Hobart, David Rog, Ben Turner, Muhammad Saif, Azza Ismail, Gavin Brittain, John A Snowden, Diana Papaioannou, Alasdair J Coles, Gordon Mazibrada, Neil Scolding, Maruthi Vinjam, Matthew Bursnall, Claire Rice, Ian Pomeroy, Majid Kazmi, Esmaeil Nikfekr, Kim Orchard, Thushan de Silva, Jennifer Petrie, Elisa Roldan, Caroline Besley, Kate E M Duffy, Rachel Glover, Katie Hullock, Colette Beecher, Ian Gabriel, Charalampia Kyriakou, Andy Peniket, Paolo A Muraro, Amy Publicover, Andy Clark, Charles Crawley, Eleni Tholouli, Fran Kinsella, Gabriele De Luca, Jeff Davies, Jenny Byrne, Keith Campbell, Leonora Finisku, Simon Bulley, and Vic Campbell
- Subjects
Medicine - Abstract
Introduction Autologous haematopoietic stem cell transplantation (aHSCT) is increasingly used as treatment for patients with active multiple sclerosis (MS), typically after failure of disease-modifying therapies (DMTs). A recent phase III trial, ‘Multiple Sclerosis International Stem Cell Transplant, MIST’, showed that aHSCT resulted in prolonged time to disability progression compared with DMTs in patients with relapsing remitting MS (RRMS). However, the MIST trial did not include many of the current high-efficacy DMTs (alemtuzumab, ocrelizumab, ofatumumab or cladribine) in use in the UK within the control arm, which are now offered to patients with rapidly evolving severe MS (RES-MS) who are treatment naïve. There remain, therefore, unanswered questions about the relative efficacy and safety of aHSCT over these high-efficacy DMTs in these patient groups. The StarMS trial (Autologous Stem Cell Transplantation versus Alemtuzumab, Ocrelizumab, Ofatumumab or Cladribine in Relapsing Remitting Multiple Sclerosis) will assess the efficacy, safety and long-term impact of aHSCT compared with high-efficacy DMTs in patients with highly active RRMS despite the use of standard DMTs or in patients with treatment naïve RES-MS.Methods and analysis StarMS is a multicentre parallel-group rater-blinded randomised controlled trial with two arms. A total of 198 participants will be recruited from 19 regional neurology secondary care centres in the UK. Participants will be randomly allocated to the aHSCT arm or DMT arm in a 1:1 ratio. Participants will remain in the study for 2 years with follow-up visits at 3, 6, 9, 12, 18 and 24 months postrandomisation. The primary outcome is the proportion of patients who achieve ‘no evidence of disease activity’ during the 2-year postrandomisation follow-up period in an intention to treat analysis. Secondary outcomes include efficacy, safety, cost-effectiveness and immune reconstitution of aHSCT and the four high-efficacy DMTs.Ethics and dissemination The study was approved by the Yorkshire and Humber—Leeds West Research Ethics Committee (20/YH/0061). Participants will provide written informed consent prior to any study specific procedures. The study results will be submitted to a peer-reviewed journal and abstracts will be submitted to relevant national and international conferences.Trial registration number ISRCTN88667898.
- Published
- 2024
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