Retrospective non-interventional study of stiripentol use in Dravet patients in the USA (Retrospective US Study - STP228)

Study type

• Study type:
  Non-interventional study
• Scope of the study:
  Drug utilisation

Study drug and medical condition

• Name of medicine:
  DIACOMIT
• Study drug International non-proprietary name (INN) or common name:
  STIRIPENTOL
• Anatomical Therapeutic Chemical (ATC) code:
  (N03AX17) stiripentol

  stiripentol
• Medical condition to be studied:
  Severe myoclonic epilepsy of infancy

Population studied

• Age groups:
  Preterm newborn infants (0 – 27 days)

  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)
• Estimated number of subjects:
  100

Study design details

• Main study objective:
  The goal of the study is to understand the management of Dravet patients who received stiripentol in the United States after the drug was made available. The main study objectives are to: • Characterize the treatment patterns of stiripentol in the US following marketing authorization • Evaluate the efficacy of stiripentol treatment on seizure frequency • Estimate the impact of stiripentol
• Outcomes:
  Frequency decreased of bilateral convulsive, Frequency of status epilepticus
• Data analysis plan:
  Statistical analyses will be descriptive