A Study Evaluating the Effectiveness of Tecfidera (Dimethyl Fumarate) on Multiple Sclerosis (MS) Disease Activity and Patient-Reported Outcomes (PROTEC)

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Biogen
ClinicalTrials.gov Identifier:
NCT01930708
First received: August 15, 2013
Last updated: June 12, 2015
Last verified: June 2015
  Purpose

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.


Condition Intervention Phase
Relapsing-Remitting Multiple Sclerosis
Multiple Sclerosis
Drug: dimethyl fumarate
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Biogen:

Primary Outcome Measures:
  • Annualized Relapse Rate (ARR) [ Time Frame: 12 Months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change from Baseline in Multiple Sclerosis Impact Scale (MSIS-29) score [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    This is a validated, 29-item, MS-specific HRQoL scale that measures the physical (20 items) and psychological (9 items) impact of MS on the participant's day-to-day life during the previous 2 weeks. For each item, the subject is asked to circle the number that best describes his or her situation. The numbers for each item range from 1 (not at all) to 5 (extremely).

  • Change from Baseline in Modified Fatigue Impact Scale-5 Item (MFIS-5) score [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    This scale consists of 5 statements that describe how fatigue may affect a person. For each statement, the participant is asked to circle the number that best indicates how often fatigue has affected him or her during the previous 4 weeks. The numbers for each question range from 0 (never) to 4 (almost always).

  • Change from Baseline in Treatment Satisfaction Questionnaire for Medication (TSQM) score [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    This is a validated, 14-item questionnaire that measures a participant's level of satisfaction/dissatisfaction with medication.

  • Change from Baseline in EQ-5D 5 level version (EQ-5D-5L) index [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    The widely validated EQ-5D includes 2 components, the EQ-5D descriptive system and the EQ VAS. The EQ-5D descriptive system provides a profile of the participant's health state in 5 dimensions (mobility, self-care, usual activities, pain/discomfort, and anxiety/depression). For each dimension, the subject is instructed to indicate whether he or she has "no problems" (level 1), "slight problems" (level 2), "moderate problems" (level 3), "severe problems (level 4), or "extreme problems/inability" (level 5) on that day. For the EQ VAS, the participant is instructed to mark an "x" on a vertical scale at the point that best describes his or her own health on that day, where 0 represents the "worst health" he or she can imagine and 100 the "best health" he or she can imagine.

  • Change from Baseline in participants-Reported Indices for Multiple Sclerosis-Activity Limitations (PRIMUS-Activity Limitations) score [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    This 15-item component of the PRIMUS assesses a participant's ability to carry out various activities of daily living during the previous week without the use of aids (e.g., cane, walker, or wheelchair) or assistance. For each item, the participant is asked whether he or she can perform the activity without difficulty or with difficulty, or is unable to perform the activity.

  • Change from Baseline in Work Productivity and Activity Impairment-Multiple Sclerosis version (WPAI-MS) score [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    This 6-item instrument assesses employment status, and, during the previous 7 days, hours of missed work due to MS or other reasons, hours worked (if employed), effect on productivity due to MS while working, and activity impairment attributable to health problems.

  • Change from Baseline in Beck Depression Inventory-Fast Screen (BDI-Fast Screen) score [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    This is a 7-item scale that evaluates depression in participants with medical illness during the prior 2 weeks. It has been validated in subjects with MS.

  • Proportion of participants with confirmed (24-week) Expanded Disability Status Scale (EDSS) progression [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    The EDSS measures disability status on a scale ranging from 0 to 10, with higher scores indicating more disability. Scoring is based on measures of impairment in eight functional systems on examination by a neurologist.

  • Annualized Relapse Rate (ARR) at Baseline (i.e., over the 12 months prior to enrollment) and at Month 6 [ Time Frame: Baseline, 6 Months ] [ Designated as safety issue: No ]
  • The proportion of participants relapsed [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
  • 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 [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
  • 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 [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
  • Proportion of participants who report taking the prescribed DMF dose [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
  • Percentage of participants who report taking the prescribed DMF dose [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
  • Reasons reported by participants for not taking prescribed DMF dose [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
  • Change from baseline in EQ Visual Analog Scale (EQVAS) score [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
    A component of the EQ-5D, where participants are asked to rate their overall health-related quality of life on a standard vertical 20 cm visual analogue scale (similar to a thermometer) between 100 (best health imaginable) and 0 (worst health imaginable).


Enrollment: 1114
Study Start Date: October 2013
Estimated Study Completion Date: March 2016
Estimated Primary Completion Date: March 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: DMF
120 mg capsule oral twice daily (BID) during the first week and 240 mg BID thereafter.
Drug: dimethyl fumarate
Administered as per the approved dosage in all countries where DMF has received marketing authorization.
Other Names:
  • Tecfidera
  • DMF
  • BG00012

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01930708

  Show 90 Study Locations
Sponsors and Collaborators
Biogen
Investigators
Study Director: Medical Director Biogen
  More Information

No publications provided

Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT01930708     History of Changes
Other Study ID Numbers: 109MS408, 2013-001656-35
Study First Received: August 15, 2013
Last Updated: June 12, 2015
Health Authority: Canada: Ethics Review Committee
Belgium: Federal Agency for Medicinal Products and Health Products
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Slovenia: Agency for Medicinal Products - Ministry of Health
Spain: Spanish Agency of Medicines
Austria: Agency for Health and Food Safety
Czech Republic: State Institute for Drug Control
Slovakia: State Institute for Drug Control
Hungary: National Institute of Pharmacy
Canada: Health Canada
Portugal: National Authority of Medicines and Health Products, IP (INFARMED)
Italy: The Italian Medicines Agency

Keywords provided by Biogen:
BG-12

Additional relevant MeSH terms:
Multiple Sclerosis
Multiple Sclerosis, Relapsing-Remitting
Sclerosis
Autoimmune Diseases
Autoimmune Diseases of the Nervous System
Demyelinating Autoimmune Diseases, CNS
Demyelinating Diseases
Immune System Diseases
Nervous System Diseases
Pathologic Processes
Dimethyl fumarate
Dermatologic Agents
Immunologic Factors
Immunosuppressive Agents
Pharmacologic Actions
Physiological Effects of Drugs
Radiation-Sensitizing Agents
Therapeutic Uses

ClinicalTrials.gov processed this record on August 31, 2015