Lambert-Eaton Myasthenic Syndrome (LEMS) Registry (LEMS-01)

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Disease epidemiology

  Effectiveness study (incl. comparative)
• Data collection methods:
  Combined primary and secondary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Observational

Study drug and medical condition

• Name of medicine:
  Firdapse
• Medical condition to be studied:
  Myasthenic syndrome

Population studied

• Short description of the study population:
  Patients diagnosed with Lambert-Eaton Myasthenic Syndrome (LEMS).
• 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:
  Lambert-Eaton Myasthenic Syndrome patients
• Estimated number of subjects:
  105

Study design details

• Main study objective:
  To obtain observational safety data (identification, frequency and severity) on patients diagnosed with LEMS. To monitor for safety signals in patients treated with Firdapse including long-term treatment.To evaluate the outcome of pregnancies in patients treated with Firdapse.To gather the same observational information from patients with LEMS not treated with Firdapse...
• Data analysis plan:
  Registry data will be analyzed as per the program’s (SAP) and will be reported periodically to the European Medicines Agency (EMA) as per post marketing commitment. Longitudinal prospective and retrospective data may be collected. Demographic and baseline characteristics will be summarized. Frequencies will be presented for the categorical variables (e.g. sex and ethnicity), and descriptive statistics will be presented for continuous variables (e.g. height, weight, and age).Periodical descriptive statistical reports and final statistical analysis may also include:Patient accrual and follow-upExposure to Firdapse (dose, duration)Clinical responsePregnancy outcomes as appropriateAdverse Events (AE) including Serious AEs.AEs will be recorded as available.