Clinical trial NCT04746976: Study of Diroximel Fumarate in the Real-World Setting
Official title: A Prospective, Observational Study Evaluating Persistence on Treatment, Safety, Tolerability, and Effectiveness of Diroximel Fumarate in the Real-World Setting (EXPERIENCE-US 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: 2021-02-10
Enrollment: 75
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 Relapsing forms of multiple sclerosis (RMS) that have been prescribed Diroximel Fumarate 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) 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 \[SAEs\]).


Eligibility criteria: 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.

Primary outcomes:
measure: Percentage of Participants on Treatment with DRF at 1 Year
timeFrame: 1 year
Secondary outcomes:
measure: Percentage of Participants on Treatment with DRF at 3 Months
timeFrame: 3 months
measure: Percentage of Participants on Treatment with DRF at 2 Years
timeFrame: 2 years
measure: Annualized Relapse Rate (ARR) with DRF
timeFrame: At 1 and 2 years
measure: Percentage of Participants Relapsed
timeFrame: At 1 and 2 years
measure: Change in Processing Speed Test (PST) Score from Baseline
timeFrame: Baseline up to 2 years
measure: Change in Score for Each Domain of Quality of Life in Neurological Disorders (Neuro- QoL™) Questionnaire
timeFrame: Baseline up to 2 years
measure: Change in Disability, as Measured by Patient Determined Disease Steps (PDDS)
timeFrame: Baseline up to 2 years
measure: Change in Work Productivity and Activity Impairment Due to Multiple Sclerosis (WPAI- MS) Scores from Baseline
timeFrame: Baseline up to 2 years
measure: Number of Participants with Gastrointestinal (GI) Adverse Events (AEs)
timeFrame: Up to 32 months
measure: Number of Participants with AEs Leading to Treatment Discontinuation
timeFrame: Up to 32 months
measure: Number of Participants with Serious Adverse Events (SAEs)
timeFrame: Up to 32 months
measure: Number of Participants categorized by the types of actions taken to mitigate GI AEs
timeFrame: Up to 32 months
measure: Median Absolute Lymphocyte Count (ALC) Over Time
timeFrame: Baseline up to 2 years
measure: Percent Change in Median ALC from Baseline
timeFrame: Baseline up to 2 years
measure: Number of Participants with Lymphopenia According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI/CTCAE)Severity Grading
timeFrame: Up to 32 months
Other outcomes:
Locations:
city: West Hollywood
country: United States
facility: Regina Berkovich MD Phd Inc
state: California
city: Washington
country: United States
facility: MedStar Georgetown University Hospital
state: District of Columbia
city: Louisville
country: United States
facility: Norton Neuroscience Institute
state: Kentucky
city: Boston
country: United States
facility: Beth Israel Deaconess Medical Center
state: Massachusetts
city: Farmington
country: United States
facility: Glendale Neurological Associates, PC
state: Michigan
city: Owosso
country: United States
facility: Memorial Healthcare
state: Michigan
city: Kansas City
country: United States
facility: St. Luke's Neurology
state: Missouri
city: Freehold
country: United States
facility: CentraState Healthcare System - Linda Cardinale MS Center
state: New Jersey
city: Livingston
country: United States
facility: MS Center at St Barnabas
state: New Jersey
city: Neptune
country: United States
facility: Multiple Sclerosis Center JSUMC
state: New Jersey
city: Buffalo
country: United States
facility: Jacobs School of Medicine and Biomedical Sciences
state: New York
city: Greensboro
country: United States
facility: Cone Health
state: North Carolina
city: Portland
country: United States
facility: Providence Brain and Spine Institute
state: Oregon
city: Danville
country: United States
facility: Geisinger Medical Center
state: Pennsylvania
city: Knoxville
country: United States
facility: Hope Neurology
state: Tennessee
city: Houston
country: United States
facility: UTHealth Neurosciences Texas Medical Center II
state: Texas
city: Newport News
country: United States
facility: Riverside Neurology Specialists
state: Virginia
city: Spokane
country: United States
facility: Providence Medical Research Center
state: Washington
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References: