Disease: Multiple Sclerosis Type of study: Interventional trial
Type of study: Interventional trial
Coordinating investigators: Øivind Torkildsen & Anne Kristine Lehmann.
Study director: Lars Bø
Patient information: on this page (Norwegian only)
Background: Autologous hematopoietic stem cell transplantation (HSCT) is a promising therapy in MS, but data from randomised clinical trials (RCTs) are limited. Haukeland University Hospital (HUH) is the national centre for such MS-therapy in Norway, and is currently conducting a multicentre, international randomised clinical trial to evaluate the efficacy and safety of autologous HSCT compared to standard high-efficacy therapies in MS. The trial is coordinated by HUH in close collaboration with centres in all Norwegian health regions and study sites in Sweden (Uppsala and Gothenburg), Denmark (Copenhagen) and the Netherlands (Amsterdam).
The objectives are to investigate the efficacy and safety of HSCT in highly active multiple sclerosis compared to standard high-efficacy therapies, and to establish sufficient evidence to support routine use of HSCT in MS.
Design: This is a randomised controlled open-label trial comparing the efficacy and safety of HSCT (n=50) compared to standard high-efficacy therapies (n=50) in highly active multiple sclerosis with breakthrough disease activity.
The primary endpoint of the study is the difference in the proportion of patients with no evidence of clinical or MRI disease activity (NEDA) after 2 years (96 weeks) in the main study, and further after 5 years (240 weeks) in the extension study.
Status: By December 2024, 96 patients have been included in the study, and enrolment will continue until the target of 100 patients is reached. In Norway, patients from all health regions in Norway are screened at the University Hospital of North Norway (Tromsø), St. Olav’s Hospital (Trondheim), Akershus University Hospital (Lørenskog), and Haukeland University Hospital (Bergen). Norwegian patients randomised for HSCT are treated at HUH, and those for standard high-efficacy MS-therapy are treated at their local hospitals. Blood sampling, imaging and clinical scoring of the Norwegian patients are performed at HUH.
Participating centres
Norway
- Haukeland University Hospital, Bergen
- Akershus University Hospital, Lørenskog
- Olav’s University Hospital, Trondheim
- University Hospital of North Norway, Tromsø
Sweden
- Sahlgrenska University Hospital, Gothenburg
- Uppsala University Hospital, Uppsala
Denmark
- Copenhagen University Hospital, Rigshospitalet
The Netherlands
- Amsterdam UMC, Amsterdam
Funding
- KLINBEFORSK
- The Regional Health Authority of Western Norway
- Haukeland University Hospital
- Participating hospitals
- The Research Council of Norway, Neuro-SysMed
- Norwegian MS Society