A Phase 3 Multicenter Trial Comparing Autologous Hematopoietic Stem Cell Transplantation to Best Available Therapy for Adults with Treatment-Resistant Relapsing Multiple Sclerosis

What this Trial Is About

Researchers are evaluating treatment options for people with relapsing multiple sclerosis MS that remains active despite current therapies. This phase 3 randomized controlled trial compares autologous hematopoietic stem cell transplantation AHSCT with the best available therapy BAT for treatment-resistant relapsing MS. MS is an immune disease that damages nerve fibers in the brain and spinal cord, causing symptoms like vision loss, weakness, sensory changes, and memory problems. The study aims to see if AHSCT better controls the disease compared to standard medicines over time. Participants will be randomly assigned to one of two groups the AHSCT group or the BAT group. The AHSCT group undergoes a three-step process including mobilization and collection of their own stem cells, high-dose chemotherapy to destroy immune cells, and reinfusion of their stored stem cells to rebuild their immune system. The BAT group will receive one of several approved medicines for relapsing MS, chosen by the study doctor. Both treatments are closely monitored throughout the study. Participants will be followed for six years after starting treatment. They will have regular assessments including brain MRI scans, neurologic exams, and blood tests to track MS relapses, disability changes, brain volume, and other health measures. Researchers will also monitor for serious side effects and infections. The main outcome is how long participants remain free from MS relapses during the first three years. This long-term follow-up aims to provide a detailed understanding of how these treatments affect disease activity and safety over time.

Best Available Therapy Versus Autologous Hematopoietic Stem Cell Transplant for Multiple Sclerosis (BEAT-MS)