A 5-year multi-center, observational post-authorization safety study to document the drug utilisation of Wakix® in the treatment of narcolepsy with or without cataplexy and to collect information on its long termthe safety of Wakix® when used in routine medical practice (WAKIX PASS)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Drug utilisation

Study drug and medical condition

• Name of medicine:
  WAKIX
• Medical condition to be studied:
  Narcolepsy

Population studied

• 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)
• Estimated number of subjects:
  300

Study design details

• Main study objective:
  To collect information on the long term safety of pitolisant Wakix® (all reported adverse events) when used in a real-life setting.
• Outcomes:
  Adverse events reported by the investigators will be coded using the MedDRA (preferred term) and assessed in terms of seriousness, severity, causality, recovery. Safety results will be described in accordance with GVP Module VI. ESS, CGI, BDI, BMI, NSAQ, 8-item Morisky, FOSQ-10, EQ5D, hospitalization, emergency visit, work day loss and drug utilization pattern : patient characteristics, medical history, co-medications, Wakix® dosage changes, Wakix® interruption and reason, co-medication changes, drop out.
• Data analysis plan:
  Quantitative variables will be described using the following descriptive statistics: sample size, number of missing values, mean, standard deviation, median, minimum and maximum. Qualitative variables will be described using the following descriptive statistics: sample size, number of missing values, and percentage of each modality calculated on the responses expressed. The bivariate statistical analyses performed will undergo statistical tests, based on the nature of the variables analyzed: For qualitative variables, the Chi2 test will be applied, apart from the theoretical sample sizes are less than 5, in this case, Yates continuity correction or Fisher's exact test will be used. For quantitative variables, when distribution is close to normal (non-significant Shapiro-Wilk test), a Student's t-test or analysis of variance will be performed. If this is not the case, non-parametric tests will be used (Wilcoxon, Kruskal-Wallis).