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A pragmatic trial to evaluate the intermediate-term effects of early, aggressive versus escalation therapy in people with multiple sclerosis
Investigator (PI): Mowry, Ellen M; Newsome, Scott
Performing Organization (PO): (Current): Johns Hopkins Medicine, Department of Neurology and Neurosurgery, Johns Hopkins Multiple Sclerosis Center / (410) 614-1522
Supporting Agency (SA): Patient-Centered Outcomes Research Institute (PCORI)
Initial Year: 2018
Final Year: 2024
Record Source/Award ID: PCORI/MS-1610-37115
Funding: Total Award Amount: $13,458,862
Award Type: Contract
Award Information: PCORI: More information and project results (when completed)
Abstract: Multiple sclerosis (MS), a top cause of neurological disability, seems to be getting more common over the past several decades. In the early phase of MS, patients have episodes (called "relapses") of problems such as trouble seeing, numbness, or weakness. Over time, the disease course changes, and people get slowly worse without improvement, often having trouble walking and ultimately requiring a wheelchair to get around. There are treatments, some stronger than others, to prevent relapses, but no treatment works once the slowly worsening phase has begun, because these two phases are caused by different processes in the nervous system. It's not even clear if using the stronger treatments early on will prevent the phase of slowly worsening disability. This question is an important one to answer, particularly since the stronger medications also come with greater and sometimes fatal risks of infections and other problems. The proposed study will evaluate if the slowly worsening phase of MS can be prevented or delayed by using stronger MS treatments up front. We will perform a randomized clinical trial of 900 people with MS. Before the trial begins, a study advisory committee, composed of people with MS, clinicians (such as neurologists), health statistics experts, and patient representative groups such as the National MS Society and the Consortium of MS Centers, will make key decisions about the trial. First, they will sort the available MS medications into two groups: higher strength versus first line. They will also determine what factors will determine if an individual patient with MS will be considered at higher risk vs. lower risk of long term disability. MS patients participating in the trial will be randomized (like a flip of a coin) to being on a higher strength versus first-line therapy. This randomization will take place separately in people at higher vs. lower risk of disability. The patients will then be followed over time to evaluate if there is a difference in whether they become more disabled if they get the stronger medications or not. We will also monitor for differences in the development of some other disabling MS symptoms, such as fatigue and cognition, and we will track the brain magnetic resonance imaging and other measures that provide clues about long term worsening. We will also evaluate, among those initially thought to be at lower risk for disability and started on a first-line treatment but who experience an incomplete response to that medication, if it's reasonable to switch to a different first-line treatment or if they need to use one of the higher strength medications to prevent later disability. This study's results will help patients and their clinician partners better understand how to treat MS to best prevent disability from occurring while at the same time not taking risks that are unnecessary by using stronger medications with some safety concerns, thus filling an important current knowledge gap.
MeSH Terms:
  • Cognition Disorders /complications
  • Disabled Persons
  • Fatigue
  • Humans
  • Infection /complications
  • Magnetic Resonance Imaging
  • Multiple Sclerosis /*drug therapy
  • Pragmatic Clinical Trials as Topic
  • Random Allocation
  • Randomized Controlled Trials as Topic
  • Recurrence
  • Risk
  • Risk Assessment
Country: United States
State: Maryland
Zip Code: 21287
UI: 20181655
Project Status: Ongoing
Record History: ('2018: Start date changed from 2017 to 2018 and project extended to 2024.',)