Non-Interventional Post-Authorisation Study to Evaluate the Safety, Tolerability and Effectiveness of Berotralstat for Patients with Hereditary Angioedema (HAE) in a Real-World Setting (APeX-N)

Study type

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

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)

Non-interventional study

• Non-interventional study design:
  Other

Study drug and medical condition

• Name of medicine:
  ORLADEYO
• Anatomical Therapeutic Chemical (ATC) code:
  (B06AC06) berotralstat

  berotralstat
• Medical condition to be studied:
  Hereditary angioedema
• Additional medical condition(s):
  Orladeyo is indicated for routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older.

Population studied

• Age groups:
  Adolescents (12 to < 18 years)

  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:
  150

Study design details

• Main study objective:
  To monitor safety and tolerability of berotralstat for routine prevention of attacks of hereditary angioedema in adult and adolescent patients during long-term administration in a real world setting
• Outcomes:
  - Incidence of adverse drug reactions (ADRs) - Duration of exposure to berotralstat, Height and weight in patients starting berotralstat (12 - 18 years of age only) Rate of patient-reported HAE attacks during observation period Rate of HAE attacks requiring treatment with standard of care medication Change in Angioedema Control Test Change in AE-QoL Number of days with HAE symptoms
• Data analysis plan:
  All safety and effectiveness variables collected in this study will be analysed descriptively. For continuous data, the mean, sample size (n), standard deviation (SD), standard error (SE), median, minimum, maximum, 25th percentile (Q1) and 75th percentile (Q3) will be provided. Categorical data will be displayed by absolute and relative frequencies (percentages). For means or proportions, 95% confidence intervals will be provided

Documents

Study publications

Lee A. Berotralstat: First Approval. Drugs. 2021 Feb;81:405-9.

Bork K, Hardt J, Witzke G. Fatal laryngeal attacks and mortality in hereditary …

Longhurst H, Cicardi M. Hereditary angio-oedema. The Lancet. 2012 Feb 4;379(981…

Aygören-Pürsün E, Bygum A, Grivcheva-Panovska V, Magerl M, Graff J, Steiner UC,…

Lumry WR, Castaldo AJ, Vernon MK, Blaustein MB, Wilson DA, Horn PT. The humanis…