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Dimethyl Fumarate (DMF) Observational Study (ESTEEM)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02047097
Recruitment Status : Recruiting
First Posted : January 28, 2014
Last Update Posted : April 1, 2020
Sponsor:
Information provided by (Responsible Party):
Biogen

Tracking Information
First Submitted Date January 24, 2014
First Posted Date January 28, 2014
Last Update Posted Date April 1, 2020
Actual Study Start Date November 30, 2013
Estimated Primary Completion Date February 28, 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: January 24, 2014)
  • The number of participants that experience Adverse Events (AEs) that lead to discontinuation of dimethyl fumarate (DMF) [ Time Frame: Up to 5 years ]
  • The number of participants that experience Serious Adverse Events (SAEs) [ Time Frame: Up to 5 years ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: July 31, 2015)
  • Duration of dimethyl fumarate (DMF) use [ Time Frame: Up to 5 years ]
  • dimethyl fumarate (DMF) dosing frequency [ Time Frame: Up to 5 years ]
  • Primary reasons for discontinuation of dimethyl fumarate (DMF) use [ Time Frame: Up to 5 years ]
  • Frequency of relapses over time [ Time Frame: Up to 5 years ]
  • Disease progression as measured by Expanded Disability Status Scale (EDSS) over time [ Time Frame: Up to 5 years ]
    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. Participants with confirmed progression of disability in EDSS physical functional system scores will be defined as those who meet one of the following criteria: an increase of ≥ 1 point from baseline system score of ≥ 1 or an increase of ≥ 2 points from baseline system score of 0 in at least 2 physical functional systems, or an increase of ≥ 2 points from baseline system score of ≥ 1 or an increase of ≥ 3 points from baseline system score of 0 in any 1 physical functional system. Worsening must be confirmed on a subsequent examination using the same criterion in the same functional system(s) at least 6 months later.
  • Multiple Sclerosis Impact Scale-29 Items (MSIS-29) physical multiple sclerosis (MS) impact score [ Time Frame: Up to 5 years ]
    The 29-item Multiple Sclerosis Impact Scale (MSIS-29) is a patient-reported outcome measure to assess the impact of MS on day-to-day life during the past 2 weeks from a patient's perspective; it measures 20 physical items and 9 psychological items. The physical score is generated by summing individual items and then transforming to a scale with a range of 0 to 100, where high scores indicate worse health.
  • Multiple Sclerosis Impact Scale-29 Items (MSIS-29) psychological multiple sclerosis (MS) impact score [ Time Frame: Up to 5 years ]
    The 29-item Multiple Sclerosis Impact Scale (MSIS-29) is a patient-reported outcome measure to assess the impact of MS on day-to-day life during the past 2 weeks from a patient's perspective; it measures 20 physical items and 9 psychological items. The physical score is generated by summing individual items and then transforming to a scale with a range of 0 to 100, where high scores indicate worse health.
  • EuroQol-5 Dimensions (5 Level) (EQ-5D-5L) index score [ Time Frame: Up to 5 years ]
    Descriptive system of health-related quality of life states consisting of five dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) each of which can take one of five responses. The responses record five levels of severity (no problems/slight problems/moderate problems/severe problems/extreme problems) within a particular EQ-5D dimension.
  • EuroQol Visual Analogue Scale (EQ VAS) (0-100 scale) [ Time Frame: Up to 5 years ]
    Standard vertical 20 cm visual analogue scale (similar to a thermometer) for recording an individual's rating for their current health-related quality of life state (often referred to as page 3 of the EQ-5D questionnaire)
  • Modified Fatigue Impact Scale-5 (MFIS-5) total score [ Time Frame: Up to 5 years ]
    MFIS-5 is a modified form of the Fatigue Impact Scale that consists of five questions that assess the impact of fatigue on physical, cognitive, and psychosocial functioning, with five response levels ranging from 0 ("Never") to 4 ("Almost always"). Total scores range from 0 to 20, with higher scores representing a greater impact of fatigue.
  • Work Productivity and Activity Impairment questionnaire: Multiple Sclerosis, Version 2.0 (WPAI-MS) impairment percentages [ Time Frame: Up to 5 years ]
    The Work Productivity and Activity Impairment (WPAI) questionnaire is a validated instrument to measure impairments in work and activities. The WPAI yields four types of scores: 1. Absenteeism (work time missed) 2. Presenteesism (impairment at work / reduced on-the-job effectiveness) 3. Work productivity loss (overall work impairment / absenteeism plus presenteeism) 4. Activity Impairment. WPAI outcomes are expressed as impairment percentages, with higher numbers indicating greater impairment and less productivity.
  • Health Care Resource Consumption Questionnaire [ Time Frame: Up to 5 years ]
    The Health Care Resource Consumption questionnaire is a survey that asks patients how MS affects their use of healthcare services and the impact it has on particular areas of their life (including number of hospitalizations [MS-related, non-MS related, relapse-related, resulting in steroid use], MS-related emergency room visits, MS-related neurologist visits, visits to other health care professionals for MS-related and other reasons).
Original Secondary Outcome Measures
 (submitted: January 24, 2014)
  • Duration of dimethyl fumarate (DMF) use [ Time Frame: Up to 5 years ]
  • dimethyl fumarate (DMF) dosing frequency [ Time Frame: Up to 5 years ]
  • Primary reasons for discontinuation of dimethyl fumarate (DMF) use [ Time Frame: Up to 5 years ]
  • Frequency of relapses over time [ Time Frame: Up to 5 years ]
  • Disease progression as measured by Expanded Disability Status Scale (EDSS) over time [ Time Frame: Up to 5 years ]
  • Multiple Sclerosis Impact Scale-29 Items (MSIS-29) physical multiple sclerosis (MS) impact score [ Time Frame: Up to 5 years ]
  • Multiple Sclerosis Impact Scale-29 Items (MSIS-29) psychological multiple sclerosis (MS) impact score [ Time Frame: Up to 5 years ]
  • EuroQol-5 Dimensions (5 Level) (EQ-5D-5L) index score [ Time Frame: Up to 5 years ]
  • EuroQol Visual Analogue Scale (EQ VAS) (0-100 scale) [ Time Frame: Up to 5 years ]
  • Modified Fatigue Impact Scale-5 (MFIS-5) total score [ Time Frame: Up to 5 years ]
  • Work Productivity and Activity Impairment questionnaire: Multiple Sclerosis, Version 2.0 (WPAI-MS) impairment percentages [ Time Frame: Up to 5 years ]
  • Health Care Consumption [ Time Frame: Up to 5 years ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Dimethyl Fumarate (DMF) Observational Study
Official Title A Multicenter, Global, Observational Study to Collect Information on Safety and to Document the Drug Utilization of Tecfidera™ (Dimethyl Fumarate) When Used in Routine Medical Practice in the Treatment of Multiple Sclerosis (ESTEEM)
Brief Summary The primary objective of the study is to determine the incidence, type, and pattern of serious adverse events (SAEs), including but not limited to infections (including opportunistic infections), hepatic events, malignancies, and renal events, and of adverse events (AEs) leading to treatment discontinuation in patients with MS treated with dimethyl fumarate (DMF). Secondary objectives of this study in this population are as follows: To determine dimethyl fumarate (DMF) prescription and utilization patterns in routine clinical practice in patients with multiple sclerosis (MS); To assess the effectiveness of dimethyl fumarate (DMF) on multiple sclerosis (MS) disease activity and disability progression in routine clinical practice as determined by the Expanded Disability Status Scale (EDSS) score and multiple sclerosis (MS) relapse information; and To assess the effect of dimethyl fumarate (DMF) on health-related quality of life, healthcare resource consumption, and work productivity.
Detailed Description Patients aged 12 to under 18 may be included where enrollment of pediatric patients in this study is considered observational and is permissible by local regulations.
Study Type Observational [Patient Registry]
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration 5 Years
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Patients undergoing routine clinical care for Multiple Sclerosis.
Condition Multiple Sclerosis
Intervention Drug: dimethyl fumarate
Provided under routine clinical care
Other Names:
  • Tecfidera
  • DMF
  • BG00012
Study Groups/Cohorts dimethyl fumarate (DMF)
Patients with multiple sclerosis receiving dimethyl fumarate (DMF) under routine clinical care
Intervention: Drug: dimethyl fumarate
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 1, 2019)
5600
Original Estimated Enrollment
 (submitted: January 24, 2014)
5000
Estimated Study Completion Date February 28, 2024
Estimated Primary Completion Date February 28, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria

Key Inclusion Criteria:

- Patients with multiple sclerosis (MS) who are newly initiating treatment with dimethyl fumarate (DMF) under routine clinical care are eligible to participate in the study.

Key Exclusion Criteria:

  • Patients with previous exposure to dimethyl fumarate (DMF), Fumaderm (fumaric acid esters), or compounded fumarates.
  • Patients participating in other clinical studies.

NOTE: Other protocol-defined inclusion/exclusion criteria may apply.

Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com
Listed Location Countries Argentina,   Australia,   Austria,   Canada,   Czechia,   Denmark,   France,   Germany,   Hungary,   Ireland,   Italy,   Netherlands,   New Zealand,   Norway,   Poland,   Portugal,   Puerto Rico,   Slovakia,   Spain,   Switzerland,   United Kingdom,   United States
Removed Location Countries Czech Republic
 
Administrative Information
NCT Number NCT02047097
Other Study ID Numbers 109MS401
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Biogen
Study Sponsor Biogen
Collaborators Not Provided
Investigators
Study Director: Medical Director Biogen
PRS Account Biogen
Verification Date March 2020