Best Available Therapy Vs Autologous Hematopoietic Stem Cell Transplant for MS (BEAT-MS)
Research type
Research Study
Full title
A Multicenter Randomized Controlled Trial of Best Available Therapy versus Autologous Hematopoietic Stem Cell Transplant for Treatment-Resistant Relapsing Multiple Sclerosis.
IRAS ID
1004366
Contact name
Richard Nicholas
Contact email
Sponsor organisation
Division of Allergy Immunology, and TransplantationNational Institute of Allergy and Infectious Disease;
Eudract number
2020-000979-21
Clinicaltrials.gov Identifier
Research summary
Multiple sclerosis (MS) is a condition affecting the brain and spinal cord causing a wide range of symptoms including problems with vision, arm or leg movement, sensation or balance. It's a lifelong condition that can cause serious disability and can reduce average life expectancy. It's mostly diagnosed in young adults and is 2-3 times more common in women than men. MS is 1 of the most common causes of disability in younger adults affecting more than 2.3 million individuals worldwide.
Over 12 disease modifying therapies (DMTs) have been approved for the treatment of relapsing forms of MS. These DMTs vary in terms of efficacy, safety, and cost. Guidelines have been published but there’s no standard treatment plan and no way to know whether a given DMT will be effective in an individual patient. There’s a significant unmet medical need in this population.
Myeloablative and immunoablative therapy followed by autologous hematopoietic stem cell transplantation (AHSCT) has shown to be effective for relapsing MS in cases where DMTs failed to control disease. AHSCT has therefore been proposed as a treatment option for patients who have failed other treatments. However, AHSCT has not been compared to contemporary high efficacy biologic DMTs in a clinical trial. Therefore, despite interest in the procedure among MS neurologists and patients, there is uncertainty on whether and when AHSCT should be given to patients. The purpose of this trial is to compare efficacy and safety of AHSCT to best available therapy (BAT) in treatment-resistant relapsing MS, to determine whether AHSCT is an appropriate treatment option for patients for whom BAT would be normally be prescribed.
This is a 6-year study with around 156 participants in the US and the UK. The study will involve physical and neurological exams, vital signs, blood tests, chest x-ray, ECG, ECHO, spirometry, questionnaires.
REC name
East of Scotland Research Ethics Service REC 2
REC reference
22/ES/0010
Date of REC Opinion
5 May 2022
REC opinion
Further Information Favourable Opinion