Study of Diroximel Fumarate in the Real-World Setting

A Prospective, Observational Study Evaluating Persistence on Treatment, Safety, Tolerability, and Effectiveness of Diroximel Fumarate in the Real-World Setting (EXPERIENCE-US Study)

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]).

Study Overview

• Status: Terminated
• Conditions: Relapsing Forms of MS
• Intervention / Treatment: Drug: Diroximel Fumarate

• Study Type: Observational
• Enrollment (Actual): 75

Contacts and Locations

Study Locations

• United States
  • California
    • West Hollywood, California, United States, 90048
      • Regina Berkovich MD PhD Inc
  • District of Columbia
    • Washington, District of Columbia, United States, 20007
      • MedStar Georgetown University Hospital
  • Kentucky
    • Louisville, Kentucky, United States, 40207
      • Norton Neuroscience Institute
  • Massachusetts
    • Boston, Massachusetts, United States, 02215
      • Beth Israel Deaconess Medical Center
  • Michigan
    • Farmington, Michigan, United States, 48071
      • Glendale Neurological Associates, PC
    • Owosso, Michigan, United States, 48867
      • Memorial Healthcare
  • Missouri
    • Kansas City, Missouri, United States, 64111
      • St. Luke's Neurology
  • New Jersey
    • Freehold, New Jersey, United States, 07728
      • CentraState Healthcare System - Linda Cardinale MS Center
    • Livingston, New Jersey, United States, 07039
      • MS Center at St Barnabas
    • Neptune, New Jersey, United States, 07753
      • Multiple Sclerosis Center JSUMC
  • New York
    • Buffalo, New York, United States, 14202
      • Jacobs School of Medicine and Biomedical Sciences
  • North Carolina
    • Greensboro, North Carolina, United States, 27405
      • Cone Health
  • Oregon
    • Portland, Oregon, United States, 97225
      • Providence Brain and Spine Institute
  • Pennsylvania
    • Danville, Pennsylvania, United States, 17822
      • Geisinger Medical Center
  • Tennessee
    • Knoxville, Tennessee, United States, 37922
      • Hope Neurology
  • Texas
    • Houston, Texas, United States, 77030
      • UTHealth Neurosciences Texas Medical Center II
  • Virginia
    • Newport News, Virginia, United States, 23601
      • Riverside Neurology Specialists
  • Washington
    • Spokane, Washington, United States, 99204
      • Providence Medical Research Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants diagnosed with Relapsing forms of multiple sclerosis (RMS) that have been prescribed Diroximel Fumarate under standard clinical care.

Description

Key Inclusion Criteria:

• 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 Exclusion Criteria:

• 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 Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Diroximel Fumarate; Participants with RMS who are receiving diroximel fumarate orally in routine clinical practice will be enrolled.	Drug: Diroximel Fumarate; As described in the arm.; Other Names: VUMERITY; BIIB098

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Percentage of Participants on Treatment with DRF at 1 Year	1 year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants on Treatment with DRF at 3 Months		3 months
Percentage of Participants on Treatment with DRF at 2 Years		2 years
Annualized Relapse Rate (ARR) with DRF		At 1 and 2 years
Percentage of Participants Relapsed		At 1 and 2 years
Change in Processing Speed Test (PST) Score from Baseline	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	Baseline up to 2 years
Change in Score for Each Domain of Quality of Life in Neurological Disorders (Neuro- QoL™) Questionnaire	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.	Baseline up to 2 years
Change in Disability, as Measured by Patient Determined Disease Steps (PDDS)	The PDDS has nine ordinal levels ranging between 0 (normal) and 8 (bedridden). Higher scores indicate greater disability.	Baseline up to 2 years
Change in Work Productivity and Activity Impairment Due to Multiple Sclerosis (WPAI- MS) Scores from Baseline	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.	Baseline up to 2 years
Number of Participants with Gastrointestinal (GI) Adverse Events (AEs)	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.	Up to 32 months
Number of Participants with AEs Leading to Treatment Discontinuation	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.	Up to 32 months
Number of Participants with Serious Adverse Events (SAEs)	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.	Up to 32 months
Number of Participants categorized by the types of actions taken to mitigate GI AEs	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.	Up to 32 months
Median Absolute Lymphocyte Count (ALC) Over Time		Baseline up to 2 years
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

Collaborators and Investigators

• Sponsor: Biogen
• Investigators: Study Director: Medical Director, Biogen

Study record dates

Study Major Dates

• Study Start (Actual): March 1, 2021
• Primary Completion (Actual): April 14, 2023
• Study Completion (Actual): April 14, 2023

Study Registration Dates

• First Submitted: February 5, 2021
• First Submitted That Met QC Criteria: February 5, 2021
• First Posted (Actual): February 10, 2021

Study Record Updates

• Last Update Posted (Actual): May 26, 2023
• Last Update Submitted That Met QC Criteria: May 24, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Keywords: Multiple Sclerosis; Clinically isolated syndrome; Active secondary progressive disease; Relapsing forms of Multiple Sclerosis
• Other Study ID Numbers: US-VUM-11760

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No