International Coordinating Investigator: Lars Bø
A randomized clinical trial for comparing autologous hematopoietic stem cell transplantation (HSCT) versus alemtuzumab in MS (The RAM-MS study)
HSCT is a promising therapy in MS, but limited data from randomized clinical trials (RCTs) are available. A Norwegian health technology assessment (HTA) confirmed this challenge, and concluded that further studies are needed (https://nyemetoder.no/metoder/autolog-stamcelletransplantasjon/). Haukeland University Hospital has been the national centre for such MS-therapy in Norway, and are currently organizing a multicentre, international randomized clinical trial to evaluate the efficacy and safety of autologous HSCT compared to standard high-efficacy therapies in MS (https://clinicaltrials.gov/ & NCT03477500). The trial is multidisciplinary and involves a close collaboration between the department of haematology (investigators Anne Kristine Lehmann and Aymen Ahmed), department of transfusion medicine and immunology (investigator Einar Kristoffersen) and the department of neurology (investigator Øivind Torkildsen).
The objective is to study whether HSCT is a safe and effective therapy in highly active multiple sclerosis compared to standard high-efficacy therapies, and to establish sufficient evidence to support routine use of HSCT in MS.
The primary endpoint of the study is the proportion of patients with no evidence of disease activity (NEDA) after 2 year (96 weeks) and further after 5 year (240 weeks).
Currently, about 45 patients have been enrolled in the study, and during the next years, more patients will be included to a maximum of n=100. Patients from all over Norway are included for screening and randomization at Haukeland University Hospital (HUS). Patients randomized for HSCT are treated at HUS, and those for standard highly active MS-therapy are treated at their local hospitals. Blood sampling, imaging and clinical scoring of the Norwegian patients are performed at HUS. The patients are primarily followed for two years and further for another 3 years in the extension phase of a total of 5 years. Patients are also recruited at centres in Sweden, Denmark and The Netherlands.