Study of Diroximel Fumarate in the Real-World Setting
- Registration Number
- NCT04746976
- Lead Sponsor
- Biogen
- Brief Summary
The primary objective of the study is to characterize the persistence to therapy in participants with relapsing forms of multiple sclerosis (RMS) treated with diroximel fumarate (DRF) in routine clinical practice. The secondary objectives of the study are to assess short-term persistence to treatment; to assess long-term persistence on treatment; to assess the effect of DRF on relapses; to assess the impact of DRF on cognition; to assess the impact of DRF on participant reported outcomes (PROs) and to explore the real-world safety profile of DRF (ie, gastrointestinal \[GI\] tolerability, lymphocyte dynamics, adverse events \[AEs\] leading to discontinuation, and serious adverse events \[SAEs\]).
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- TERMINATED
- Sex
- All
- Target Recruitment
- 75
- Have a diagnosis of MS and satisfy the approved therapeutic indication for DRF per the USPI.
- DRF prescribed and planned to be initiated within 60 days after enrollment.
Key
- History of gastric bypass or required use of feeding tubes.
- Have received prior treatment with DRF.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Diroximel Fumarate Diroximel Fumarate Participants with RMS who are receiving diroximel fumarate orally in routine clinical practice will be enrolled.
- Primary Outcome Measures
Name Time Method Percentage of Participants on Treatment with DRF at 1 Year 1 year
- Secondary Outcome Measures
Name Time Method Percentage of Participants on Treatment with DRF at 3 Months 3 months Change in Processing Speed Test (PST) Score from Baseline Baseline up to 2 years PST measures mental processing speed. The PST will be assessed using multiple sclerosis performance test (MSPT). The participants will be shown some symbols and corresponding numbers. The participants will be then asked to input the numbers corresponding to the given symbols in empty rows. Higher number of correct responses indicates better cognition
Change in Disability, as Measured by Patient Determined Disease Steps (PDDS) Baseline up to 2 years The PDDS has nine ordinal levels ranging between 0 (normal) and 8 (bedridden). Higher scores indicate greater disability.
Percentage of Participants on Treatment with DRF at 2 Years 2 years Change in Score for Each Domain of Quality of Life in Neurological Disorders (Neuro- QoL™) Questionnaire Baseline up to 2 years Neuro-QOL uses a T score which has a mean of 50 and SD of 10, based on the norming sample used. All Neuro-QOL banks and scales are scored such that a high score reflects more of what is being measured.
Annualized Relapse Rate (ARR) with DRF At 1 and 2 years Percentage of Participants Relapsed At 1 and 2 years Number of Participants categorized by the types of actions taken to mitigate GI AEs Up to 32 months The actions taken will include temporary dose reduction, temporary dose interruption, initiation of concomitant GI medication, taking DRF dose with food, permanent discontinuation, or other action.
Change in Work Productivity and Activity Impairment Due to Multiple Sclerosis (WPAI- MS) Scores from Baseline Baseline up to 2 years The Work Productivity and Activity Impairment (WPAI) questionnaire is a validated instrument to measure impairments in work and activities. The WPAI yields four types of scores: 1. Absenteeism (work time missed) 2. Presenteesism (impairment at work / reduced on-the-job effectiveness) 3. Work productivity loss (overall work impairment / absenteeism plus presenteeism) 4. Activity Impairment. Higher numbers indicate greater impairment and less productivity.
Number of Participants with Serious Adverse Events (SAEs) Up to 32 months An SAE is any untoward medical occurrence that at any dose results in death, in the view of the investigator, places the participant at immediate risk of death, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect or is a medically important event.
Percent Change in Median ALC from Baseline Baseline up to 2 years Number of Participants with Lymphopenia According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI/CTCAE)Severity Grading Up to 32 months Number of Participants with Gastrointestinal (GI) Adverse Events (AEs) Up to 32 months An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Number of Participants with AEs Leading to Treatment Discontinuation Up to 32 months An AE is any untoward medical occurrence in a participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal assessment such as an abnormal laboratory value), symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.
Median Absolute Lymphocyte Count (ALC) Over Time Baseline up to 2 years
Trial Locations
- Locations (18)
Beth Israel Deaconess Medical Center
🇺🇸Boston, Massachusetts, United States
MS Center at St Barnabas
🇺🇸Livingston, New Jersey, United States
Memorial Healthcare
🇺🇸Owosso, Michigan, United States
Regina Berkovich MD Phd Inc
🇺🇸West Hollywood, California, United States
MedStar Georgetown University Hospital
🇺🇸Washington, District of Columbia, United States
Norton Neuroscience Institute
🇺🇸Louisville, Kentucky, United States
St. Luke's Neurology
🇺🇸Kansas City, Missouri, United States
Jacobs School of Medicine and Biomedical Sciences
🇺🇸Buffalo, New York, United States
Providence Brain and Spine Institute
🇺🇸Portland, Oregon, United States
UTHealth Neurosciences Texas Medical Center II
🇺🇸Houston, Texas, United States
Geisinger Medical Center
🇺🇸Danville, Pennsylvania, United States
Hope Neurology
🇺🇸Knoxville, Tennessee, United States
Riverside Neurology Specialists
🇺🇸Newport News, Virginia, United States
Providence Medical Research Center
🇺🇸Spokane, Washington, United States
Cone Health
🇺🇸Greensboro, North Carolina, United States
CentraState Healthcare System - Linda Cardinale MS Center
🇺🇸Freehold, New Jersey, United States
Glendale Neurological Associates, PC
🇺🇸Farmington, Michigan, United States
Multiple Sclerosis Center JSUMC
🇺🇸Neptune, New Jersey, United States