A Multinational Phase IV Study Evaluating “Real World” Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog alfa) for Routine Prophylaxis (TAURUS)

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

  Drug utilisation

  Effectiveness study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Open label, prospective, non-interventional, single arm study in patients receiving KOVALTRY as prophylaxis therapy.

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (B02BD02) coagulation factor VIII

  coagulation factor VIII
• Medical condition to be studied:
  Coagulation factor VIII level decreased

Population studied

• Short description of the study population:
  Previously treated male patients with moderate to severe (≤ 5% FVIII:C) hemophilia A, with ≥ 50 exposure days (EDs) to any FVIII product and with or without history of inhibitors who have been prescribed KOVALTRY for a medically appropriate use will be eligible to be included into this study. Indications and contraindications according to the local market authorization should carefully be considered.; Inclusion criterion/criteria; • Male patients diagnosed with moderate to severe hemophilia A (≤ 5% FVIII:C); • Any age; • ≥ 50 exposure days (EDs) to any FVIII product; • Patients with or without history of inhibitors; • Patient with previous history of inhibitors, with at least 2 consecutive negative inhibitor tests and on standard prophylaxis therapy for at least 1 year prior to study entry; • No current evidence* of FVIII inhibitor or clinical suspicion** of FVIII inhibitor; *Evidence of FVIII inhibitor as measured by the Nijmegen-modified Bethesda assay[<0.6 Bethesda units (BU/mL)] or Bethesda assay [< 1.0 BU/mL] in 2 on consecutives samples; **Documented or clinical suspicion of shortened FVIII half-life (< 6 hrs); • Currently on or plan to start prophylaxis therapy with KOVALTRY; • Written informed consent; Exclusion criterion/criteria; • Patients participating in an investigational program with interventions outside of routine clinical practice; • Patients with an additional diagnosis of any bleeding/coagulation disorder other than hemophilia A; • Patients on Immune Tolerance Induction (ITI) treatment at the time of enrollment
• Age groups:
  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  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)
• Special population of interest:
  Other
• Special population of interest, other:
  Patients with moderate to severe Hemophilia A
• Estimated number of subjects:
  350

Study design details

• Main study objective:
  The primary objective of this study is to investigate weeklyprophylaxis dosing regimens used in standard clinicalpractice.
• Outcomes:
  Proportion of patients on 2x weekly prophylaxis,Proportion of patients on 3x weekly prophylaxis. Composite number of reported bleeds (total, spontaneous, joint and trauma),Number of physicians who selected the individual criteria as top 3 reason for the prophylaxis decision,Mean and median composite score for treatment satisfaction (Hemo-SAT),Incidence of adverse events (AEs) and serious adverse events (SAEs).
• Data analysis plan:
  Statistical analyses will be explorative. All variables will be analyzed descriptively by frequency tables and/or summary statistics. Changes from baseline will be provided.