Clinical trial NCT01930708: A Study Evaluating the Effectiveness of Tecfidera (Dimethyl Fumarate) on Multiple Sclerosis (MS) Disease Activity and Patient-Reported Outcomes
Official title: A Multicenter, Open-Label Study Evaluating the Effectiveness of Oral Tecfidera™ (Dimethyl Fumarate) on MS Disease Activity and Patient-Reported Outcomes in Subjects With Relapsing-Remitting Multiple Sclerosis in the Real-World Setting
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Relapsing-Remitting Multiple Sclerosis
Multiple Sclerosis
Keywords: BG-12
Lead sponsor: Biogen
Sponsor class: INDUSTRY
First posted: 2013-08-29
Enrollment: 1114
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of the study is to estimate the annualized relapse rate (ARR) in participants with Relapsing Remitting Multiple Sclerosis (RRMS) who are treated with dimethyl fumarate (DMF) over a 12-month period.

The secondary objectives of this study in this population are to assess the impact of DMF over a 12-month period on participants -reported health-related quality of life (HRQoL) outcomes, additional clinical effectiveness outcomes, and health economics-related outcomes, and to characterize participants-reported adherence to DMF.


Eligibility criteria: Key Inclusion Criteria:

* Have a diagnosis of Relapsing-Remitting Multiple Sclerosis (RRMS) and satisfy the approved therapeutic indication for DMF (per the local DMF product information).
* Must be naïve to DMF, Fumaderm®, and other compounded fumarates, and to MS therapies that are primarily prescribed second-line (e.g., natalizumab, fingolimod) and to alemtuzumab.
* Have a recent complete blood count (CBC) that does not preclude the subject's participation in the study, in the judgment of the Investigator.

Key Exclusion Criteria:

* Are unwilling or unable to comply with study requirements, or are deemed unsuitable for study participation as determined by the Investigator.
* Have major comorbid conditions that preclude participation in the study, as determined by the Investigator.
* Are pregnant, unless DMF is clearly needed and the potential benefit of DMF to the subjects justifies the potential risk to the fetus, in the judgment of the Investigator (in all countries except Austria). In Austria, pregnant subjects are excluded from participation in the study.
* Are women of childbearing potential and are not using appropriate contraception (per the local DMF product information) as determined by the Investigator.
* Women who are breastfeeding may be excluded (per the local DMF product information) at the discretion of the Investigator.
* Have previously received or are receiving treatment with MS therapies primarily used second-line (e.g., natalizumab, fingolimod) or alemtuzumab, or are currently receiving and planning to continue on other disease-modifying therapies for RRMS.
* Are hypersensitive to the active ingredient in the DMF drug product (i.e., DMF) or to any of the excipients listed in the local DMF product information.
* Current enrollment in any clinical trial except for the Biogen Idec DMF Pregnancy Exposure Registry or other studies that, according to the study Medical Director, do not conflict with this study (e.g., health economics studies or local registries).

Other protocol-defined inclusion/exclusion criteria may apply.

Primary outcomes:
measure: Annualized Relapse Rate (ARR)
timeFrame: 12 Months
Secondary outcomes:
measure: Change from Baseline in Multiple Sclerosis Impact Scale (MSIS-29) score
timeFrame: 12 Months
measure: Change from Baseline in Modified Fatigue Impact Scale-5 Item (MFIS-5) score
timeFrame: 12 Months
measure: Change from Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) score
timeFrame: 12 Months
measure: Change from Baseline in EQ-5D 5 level version (EQ-5D-5L) index
timeFrame: 12 Months
measure: Change from Baseline in participants-Reported Indices for Multiple Sclerosis-Activity Limitations (PRIMUS-Activity Limitations) score
timeFrame: 12 Months
measure: Change from Baseline in Work Productivity and Activity Impairment-Multiple Sclerosis version (WPAI-MS) score
timeFrame: 12 Months
measure: Change from Baseline in Beck Depression Inventory-Fast Screen (BDI-Fast Screen) score
timeFrame: 12 Months
measure: Proportion of participants with confirmed (24-week) Expanded Disability Status Scale (EDSS) progression
timeFrame: 12 Months
measure: Annualized Relapse Rate (ARR) at Baseline (i.e., over the 12 months prior to enrollment) and at Month 6
timeFrame: Baseline, 6 Months
measure: The proportion of participants relapsed
timeFrame: 12 Months
measure: Number of participants who are hospitalized/have emergency room visits due to MS relapses or have relapses requiring intravenous (IV) steroid treatment during the study, or who make visits to neurologists/other specialists due to MS
timeFrame: 12 Months
measure: Proportion of participants who are hospitalized/have emergency room visits due to MS relapses or have relapses requiring intravenous (IV) steroid treatment during the study, or who make visits to neurologists/other specialists due to MS
timeFrame: 12 Months
measure: Proportion of participants who report taking the prescribed DMF dose
timeFrame: 12 Months
measure: Percentage of participants who report taking the prescribed DMF dose
timeFrame: 12 Months
measure: Reasons reported by participants for not taking prescribed DMF dose
timeFrame: 12 Months
measure: Change from baseline in EQ Visual Analog Scale (EQVAS) score
timeFrame: 12 Months
Other outcomes:
Locations:
city: Innsbruck
country: Austria
facility: Research Site
city: Klagenfurt
country: Austria
facility: Research Site
city: Linz
country: Austria
facility: Research Site
city: Salzburg
country: Austria
facility: Research Site
city: St. Polten
country: Austria
facility: Research Site
city: Villach
country: Austria
facility: Research Site
city: Wien
country: Austria
facility: Research Site
city: Wien
country: Austria
facility: Research Site
city: Brugge
country: Belgium
facility: Research Site
city: Bruxelles
country: Belgium
facility: Research Site
city: Melsbroek
country: Belgium
facility: Research Site
city: Calgary
country: Canada
facility: Research Site
state: Alberta
city: Victoria
country: Canada
facility: Research Site
state: British Columbia
city: St. John's
country: Canada
facility: Research Site
state: New Brunswick
city: St. John's
country: Canada
facility: Research Site
state: Newfoundland and Labrador
city: Halifax
country: Canada
facility: Research Site
state: Nova Scotia
city: London
country: Canada
facility: Research Site
state: Ontario
city: Ottawa
country: Canada
facility: Research Site
state: Ontario
city: Montreal
country: Canada
facility: Research Site
state: Quebec
city: Brno
country: Czechia
facility: Research Site
city: Havirov
country: Czechia
facility: Research Site
city: Hradec Kralove
country: Czechia
facility: Research Site
city: Olomouc
country: Czechia
facility: Research Site
city: Pardubice
country: Czechia
facility: Research Site
city: Praha 10
country: Czechia
facility: Research Site
city: Praha 2
country: Czechia
facility: Research Site
city: Strasbourg Cedex
country: France
facility: Research Site
state: Bas Rhin
city: Dijon Cedex
country: France
facility: Research Site
state: Cote dÝOr
city: Besancon cedex
country: France
facility: Research Site
state: Doubs
city: Nimes
country: France
facility: Research Site
state: Gard
city: Bordeaux Cedex
country: France
facility: Research Site
state: Gironde
city: Colmar cedex
country: France
facility: Research Site
state: Haut Rhin
city: Toulouse cedex 1
country: France
facility: Research Site
state: Haute Garonne
city: Limoges cedex
country: France
facility: Research Site
state: Haute Vienne
city: Rennes cedex 09
country: France
facility: Research Site
state: Ille Et Vilaine
city: Grenoble cedex 09
country: France
facility: Research Site
state: Isere
city: Nantes cedex 1
country: France
facility: Research Site
state: Loire Atlantique
city: Reims
country: France
facility: Research Site
state: Marne
city: Lille Cedex
country: France
facility: Research Site
state: Nord
city: Lille Cedex
country: France
facility: Research Site
state: Nord
city: La Roche sur Yon
country: France
facility: Research Site
state: Vendee
city: Poitiers cedex
country: France
facility: Research Site
state: Vienne
city: Le Chesnay Cedex
country: France
facility: Research Site
state: Yvelines
city: Nancy
country: France
facility: Research Site
city: Rouen Cedex
country: France
facility: Research Site
city: Budapest
country: Hungary
facility: Research Site
city: Budapest
country: Hungary
facility: Research Site
city: Debrecen
country: Hungary
facility: Research Site
city: Pecs
country: Hungary
facility: Research Site
city: Szeged
country: Hungary
facility: Research Site
city: Veszprem
country: Hungary
facility: Research Site
city: Ancona
country: Italy
facility: Research Site
city: Bergamo
country: Italy
facility: Research Site
city: Bolzano
country: Italy
facility: Research Site
city: Castelfiorentino
country: Italy
facility: Research Site
city: Firenze
country: Italy
facility: Research Site
city: Genova
country: Italy
facility: Research Site
city: Messina
country: Italy
facility: Research Site
city: Milano
country: Italy
facility: Research Site
city: Napoli
country: Italy
facility: Research Site
city: Perugia
country: Italy
facility: Research Site
city: Pozzilli
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Roma
country: Italy
facility: Research Site
city: Siena
country: Italy
facility: Research Site
city: Torino
country: Italy
facility: Research Site
city: Almada
country: Portugal
facility: Research Site
city: Amadora
country: Portugal
facility: Research Site
city: Guimarães
country: Portugal
facility: Research Site
city: Lisboa
country: Portugal
facility: Research Site
city: Lisboa
country: Portugal
facility: Research Site
city: Loures
country: Portugal
facility: Research Site
city: Porto
country: Portugal
facility: Research Site
city: Porto
country: Portugal
facility: Research Site
city: Setúbal
country: Portugal
facility: Research Site
city: Bratislava
country: Slovakia
facility: Research Site
city: Presov
country: Slovakia
facility: Research Site
city: Ljubljana
country: Slovenia
facility: Research Site
city: Maribor
country: Slovenia
facility: Research Site
city: Majadahonda
country: Spain
facility: Research Site
state: Madrid
city: Barcelona
country: Spain
facility: Research Site
city: Bilbao
country: Spain
facility: Research Site
city: Córdoba
country: Spain
facility: Research Site
city: El Palmar
country: Spain
facility: Research Site
city: Girona
country: Spain
facility: Research Site
city: Malaga
country: Spain
facility: Research Site
city: Sevilla
country: Spain
facility: Research Site
city: Valencia
country: Spain
facility: Research Site
city: Vigo
country: Spain
facility: Research Site
city: Zaragoza
country: Spain
facility: Research Site
Study officials:
affiliation: Biogen
name: Medical Director
role: STUDY_DIRECTOR
References:
citation: Berger T, Brochet B, Brambilla L, Giacomini PS, Montalban X, Vasco Salgado A, Su R, Bretagne A. Effectiveness of delayed-release dimethyl fumarate on patient-reported outcomes and clinical measures in patients with relapsing-remitting multiple sclerosis in a real-world clinical setting: PROTEC. Mult Scler J Exp Transl Clin. 2019 Dec 2;5(4):2055217319887191. doi: 10.1177/2055217319887191. eCollection 2019 Oct-Dec.