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)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Prospective, global, observational study

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  DIMETHYL FUMARATE
• Medical condition to be studied:
  Multiple sclerosis

Population studied

• Short description of the study population:
  The study population included patients aged 18 years or older diagnosed with multiple sclerosis (MS) newly prescribed with treatment of dimethyl fumarate (DMF) under routine clinical practice. ; Inclusion criteria:; - Patients must be naïve to DMF, Fumaderm®, and compounded fumarates at the time of enrollment, but need not be naïve to other MS treatments, and must not be currently enrolled in any other clinical trial or study except for the DMF Pregnancy Registry or other studies that, according to the study Medical Director, do not conflict with this observational study (e.g., health economic studies). ; ; Exclusion criteria:; - Patients with previous exposure to DMF, Fumaderm®, and compounded fumarates are excluded so as not to introduce bias; these patients may be more or less likely to experience AEs and may fail to report AEs that occurred before study enrollment. ; - Patients participating in other clinical studies are excluded so as not to unduly confound causality assessments when a concomitant experimental agent’s safety profile has yet to be established and/or the physician is blinded to the patient’s treatment assignment.
• Age groups:
  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with multiple sclerosis
• Estimated number of subjects:
  5496

Study design details

• Main study objective:
  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 Multiple Sclerosis (MS) treated with dimethyl fumarate (DMF).
• Outcomes:
  The number of participants that experience Adverse Events (AEs) that lead to discontinuation of dimethyl fumarate (DMF) and the number of participants that experience Serious Adverse Events (SAEs). DMF prescription and utilization patterns, Effectiveness of DMF on MS disease activity and disability progression, Changes in health-related quality of life measures will be evaluated over time.
• Data analysis plan:
  Statistical analyses will be based on all patients who enroll in the study and take at least 1 dose of DMF. Statistical analyses will generally be descriptive and exploratory in nature. No formal statistical hypothesis testing is planned. Ninety-five percent CIs for incidence and incidence rate point estimates will be calculated using the binomial distribution and the Poisson distribution, respectively. Analyses of clinical laboratory parameters may include summaries of actual values over time, change from baseline over time, percent change from baseline over time, shift tables, and/or summaries of worst post-baseline values. Annualized relapse rate will be analyzed using a negative binomial model, adjusted for appropriate prognostic factors, and time-to-event endpoints will be analyzed using Kaplan-Meier estimates. Summary statistics will be presented for health-related quality of life, healthcare resource consumption, and work productivity outcomes over time.

Documents

Study results

109MS401_CSR Synopsis 21Aug2023_Redacted