Exploring Diroximel Fumarate Real-world Experience in Canada and Israel (EXPER-CA/IL)

A Prospective, Observational Study Evaluating Persistence on Treatment, Safety, Tolerability, and Effectiveness of Diroximel Fumarate in the Real-World Setting (EXPERIENCE-CA+IL 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); to assess the impact of DRF on disability; to assess treatment satisfaction with DRF; to explore the real-world safety profile of DRF (i.e., 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): 64

Contacts and Locations

Study Locations

• Israel
  • Haifa, Israel
    • Lady Davis Carmel Medical Center
  • Jerusalem, Israel
    • Hadassah Medical Center
  • Kfar Saba, Israel
    • Meir Medical Center
  • Petah Tikva, Israel
    • Rabin Medical Center
  • Tel-Aviv, Israel
    • Tel Aviv Sourasky Medical
  • Tel-Hashomer, Israel
    • Sheba Medical 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 RMS that have been prescribed DRF under standard clinical care.

Description

Key Inclusion Criteria:

• Have a diagnosis of MS and satisfy the approved therapeutic indication for DRF per the prescribing information.
• DRF prescribed and planned to be initiated within 60 days of enrollment or already initiated, with enrollment occurring no more than 7 days since the first dose.

Key Exclusion Criteria:

• History of gastric bypass or required use of feeding tubes.
• Current enrollment in any interventional study or in any study which may conflict with this study, per the discretion of the principal investigator (PI) and Biogen
• Have received prior treatment with DRF (more than 7 days before enrollment).

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Diroximel Fumarate (DRF); Participants with a confirmed diagnosis of MS who are newly prescribed DRF in routine clinical practice and who satisfy the approved therapeutic indication for DRF will be enrolled.	Drug: Diroximel Fumarate; Administered as specified in the treatment arm.; Other Names: VUMERITY; BIIB098

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Percentage of Participants on Treatment with DRF		Month 3
Percentage of Participants on Treatment with DRF		Year 2
Annualized Relapse Rate (ARR) with DRF	For ARR at 1 year, the total number of days on study will be defined as the number of days since date of first dose to 1 year (365 days) if participant stay on DRF treatment for longer than 1 year, or the date of first dose to the date of DRF discontinuation if participant die or withdraw from the study prior to 1 year. For ARR at 2 years, the total number of days on study is defined as the number of days since the date of first dose to the date of DRF discontinuation. The relapse rate will be calculated as the total number of relapses experienced divided by the total number of days on DRF treatment, and the ratio multiplied by 365.	Year 1 and 2
Percentage of Participants Relapsed		Year 1 and 2
Change in Cognitive Processing Speed Test (CPST) Score	Change in CPST will be assessed using the Konectom application. Cognitive function will be evaluated based on elements of attention, psychomotor speed, visual processing and working memory. Cognitive function is assessed by having the participant pair abstract symbols with specific numbers using a key as a guide. Higher number of correct responses indicates better cognition	Baseline, Year 1 and 2
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, Year 1 and 2
Change in Disability, as Measured by Expanded Disability Status Scale (EDSS)	The EDSS measures disability status on a scale ranging from 0 (normal) to 10 (death due to multiple sclerosis [MS]), with higher scores indicating more disability. Scoring is based on measures of impairment in eight functional systems on examination by a neurologist.	Baseline, Year 1 and 2
Mean Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Score	TSQM Version 1.4 is comprised of 14 questions that provide scores on four scales: effectiveness (3 items), side effects (5 items), convenience (3 items) and global satisfaction (3 items). The scale scores are transformed and range from 0 to 100. Higher scores indicate greater satisfaction.	Baseline, Year 1 and 2
Number of Participants with Gastrointestinal (GI) Adverse Events (AEs)	An AE is any untoward medical occurrence in a participant or clinical investigation 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, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.	Up to Month 32
Number of Participants with AEs Leading to Treatment Discontinuation	An AE is any untoward medical occurrence in a participant or clinical investigation 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, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether or not related to the medicinal (investigational) product.	Up to Month 32
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 (a life-threatening event), 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 Month 32
Number of Participants with Relevant Concomitant Medication Use	The concomitant medication may include GI symptom medication, coronavirus disease of 2019 (COVID-19) vaccine, etc.	Up to Month 32
Number of Participants Categorized by the Types of Actions Taken Due to 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 Month 32
Median Absolute Lymphocyte Count (ALC) Over Time		Baseline, Month 6, Year 1 and 2
Percent Change from Baseline in Median ALC		Baseline, Month 6, Year 1 and 2
Number of Participants with Lymphopenia According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI/CTCAE) Severity Grading		Up to Month 32

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Actual): December 9, 2021
• Primary Completion (Actual): April 14, 2023
• Study Completion (Actual): April 14, 2023

Study Registration Dates

• First Submitted: June 28, 2021
• First Submitted That Met QC Criteria: June 28, 2021
• First Posted (Actual): July 2, 2021

Study Record Updates

• Last Update Posted (Actual): May 10, 2023
• Last Update Submitted That Met QC Criteria: May 9, 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: CA-VUM-11892

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