C1 inhibitor Treatment Registry to assess the Safety and Immunological Profile of Ruconest in the treatment of HAE Attacks (Ruconest Registry)

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:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prescription event monitoring

Study drug and medical condition

• Name of medicine:
  RUCONEST
• Study drug International non-proprietary name (INN) or common name:
  CONESTAT ALFA
• Anatomical Therapeutic Chemical (ATC) code:
  (B06AC) Drugs used in hereditary angioedema

  Drugs used in hereditary angioedema

  (B06AC04) conestat alfa

  conestat alfa
• Medical condition to be studied:
  Hereditary angioedema

Population studied

• Age groups:
  Children (2 to < 12 years)

  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:
  300

Study design details

• Main study objective:
  To observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest in patients diagnosed with HAE
• Outcomes:
  Incidence of AEs reported per patient after single or repeated treatment with Ruconest or plasma-derived C1 inhibitor, Incidence of positive immunological test findings after treatment with Ruconest
• Data analysis plan:
  Incidences of patients with ADRs (globally, on preferred term and system organ class level) will be calculated for each Safety Analysis Set, 2-sided 95% confidence intervals will also be given. ; In addition, incidences of patients with ADRs for each treatment number as well as overall percentages of treatments with ADRs will be given.; The number of treatments will be counted by patient and described by frequencies for each Safety Analysis Set. ; The mean time between treatments (i.e. ratio of time under observation relative to number of treatments) will be described on a metric scale.; Efficacy will be described by frequencies for the overall assessment.; The statistical analysis will be descriptive and consist of frequency distributions (frequencies and percentages) for categorical variables, and descriptive statistics (mean, median, standard deviation, minimum, maximum, and number of observations) for quantitative variables, given for each Safety Analysis Set.

Documents

Study results

C1 1412 Ruconest Registry Report summary of results version 1.0 08APR2025.pdf