Clinical trial NCT04948606: Exploring Diroximel Fumarate Real-world Experience in Canada and Israel
Official title: A Prospective, Observational Study Evaluating Persistence on Treatment, Safety, Tolerability, and Effectiveness of Diroximel Fumarate in the Real-World Setting (EXPERIENCE-CA+IL Study)
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Relapsing Forms of MS
Keywords: Clinically isolated syndrome
Multiple Sclerosis
Relapsing forms of Multiple Sclerosis
Active secondary progressive disease
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2021-07-02
Enrollment: 64
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants diagnosed with RMS that have been prescribed DRF under standard clinical care.

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); 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 \[SAEs\].


Eligibility criteria: 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.

Primary outcomes:
measure: Percentage of Participants on Treatment with DRF
timeFrame: Year 1
Secondary outcomes:
measure: Percentage of Participants on Treatment with DRF
timeFrame: Month 3
measure: Percentage of Participants on Treatment with DRF
timeFrame: Year 2
measure: Annualized Relapse Rate (ARR) with DRF
timeFrame: Year 1 and 2
measure: Percentage of Participants Relapsed
timeFrame: Year 1 and 2
measure: Change in Cognitive Processing Speed Test (CPST) Score
timeFrame: Baseline, Year 1 and 2
measure: Change in Score for Each Domain of Quality of Life in Neurological Disorders (Neuro- QoL™) Questionnaire
timeFrame: Baseline, Year 1 and 2
measure: Change in Disability, as Measured by Expanded Disability Status Scale (EDSS)
timeFrame: Baseline, Year 1 and 2
measure: Mean Change in Treatment Satisfaction Questionnaire for Medication (TSQM) Score
timeFrame: Baseline, Year 1 and 2
measure: Number of Participants with Gastrointestinal (GI) Adverse Events (AEs)
timeFrame: Up to Month 32
measure: Number of Participants with AEs Leading to Treatment Discontinuation
timeFrame: Up to Month 32
measure: Number of Participants with Serious Adverse Events (SAEs)
timeFrame: Up to Month 32
measure: Number of Participants with Relevant Concomitant Medication Use
timeFrame: Up to Month 32
measure: Number of Participants Categorized by the Types of Actions Taken Due to GI AEs
timeFrame: Up to Month 32
measure: Median Absolute Lymphocyte Count (ALC) Over Time
timeFrame: Baseline, Month 6, Year 1 and 2
measure: Percent Change from Baseline in Median ALC
timeFrame: Baseline, Month 6, Year 1 and 2
measure: Number of Participants with Lymphopenia According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI/CTCAE) Severity Grading
timeFrame: Up to Month 32
Other outcomes:
Locations:
city: Haifa
country: Israel
facility: Lady Davis Carmel Medical Center
city: Jerusalem
country: Israel
facility: Hadassah Medical Center
city: Kfar Saba
country: Israel
facility: Meir Medical Center
city: Petah Tikva
country: Israel
facility: Rabin Medical Center
city: Tel-Aviv
country: Israel
facility: Tel Aviv Sourasky Medical
city: Tel-Hashomer
country: Israel
facility: Sheba Medical Center
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: