NN1841-3868 Use of rFXIII in treatment of congenital FXIII deficiency, a prospective multi-centre observational study (mentor™6)

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prospective, single-arm, multi-centre post-authorisation safety study (PASS)

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  CATRIDECACOG
• Medical condition to be studied:
  Factor XIII deficiency

Population studied

• Short description of the study population:
  This non-interventional study will include patients with congenital FXIII A-subunit deficiency for whom the decision to treat with rFXIII has been made and who are willing to provide informed consent (or patient’s legally acceptable representative (LAR) consent, if applicable). The study will aim at observing all patients exposed to rFXIII in the EU, and additional patients from selected nonEU countries. The study will run for 5 years, where after data will be reported and the study closed.; Inclusion criteria; 1. Informed consent obtained before any study-related activities. (Study-related activities are any procedure related to recording of data according to the protocol).; 2. Able and willing to provide signed informed consent (or patient’s legally acceptable representative (LAR) consent, if applicable), as required by local ethics committee, governmental or regulatory authorities.; 3. Congenital FXIII A-subunit deficiency.; 4. Actual or planned exposure to the rFXIII.; Exclusion criteria; 1. Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
• Age groups:
  • Paediatric Population (< 18 years)

    • Adolescents (12 to < 18 years)
    • Children (2 to < 12 years)
    • Neonate

      • Preterm newborn infants (0 – 27 days)
  • Adult and elderly population (≥18 years)

    • Adults (18 to < 65 years)

      • Adults (18 to < 46 years)
      • Adults (46 to < 65 years)
    • Elderly (≥ 65 years)

      • Adults (65 to < 75 years)
• Special population of interest:
  Other
• Special population of interest, other:
  Congenital FXIII deficiency patients
• Estimated number of subjects:
  30

Study design details

• Main study objective:
  The aim of this observational study is to investigate the incidence of specific adverse drug reactions associated with the use of recombinant factor XIII (rFXIII) in patients with congenital FXIII A-subunit deficiency, comprising FXIII antibodies, allergic reactions, embolic and thrombotic events and lack of therapeutic effect.
• Outcomes:
  Adverse drug reactions in patients with congenital FXIII A-subunit deficiency treated with rFXIII, comprising FXIII antibodies, allergic reactions, embolic and thrombotic events and lack of effect, collected during study period up to 6 years. - All serious adverse events collected- All medical events of special interest collected- All medication errors and near medication errors collected- Use of rFXIII in patients with congenital FXIII A-subunit deficiency also for other uses than for prophylactic treatment collected- Annualised bleeding rate- All outcomes are collected during study period up to 6 years
• Data analysis plan:
  This is a purely descriptive study and the statistical analyses and presentations do not include any testing of pre-specified hypotheses.