Hemophilia A, Congenital
Conditions
Keywords
Previously treated patients
Brief summary
The primary objective of this study is to investigate weekly prophylaxis dosing regimens used in standard clinical practice. In addition the study will capture reported bleed rate, pattern of change in KOVALTRY prophylaxis dose & dosing frequency, reason for choice of treatment regimen, FVIII product switch pattern, patient treatment satisfaction and adherence, KOVALTRY pharmacokinetic data (if performed), KOVALTRY consumption, as well as safety data.
Detailed description
Open label, prospective, non-interventional, single arm study in patients receiving KOVALTRY as prophylaxis therapy.
Interventions
BIOLOGICALKovaltry (Antihemophilic Factor [Recombinant], BAY81-8973
unmodified, full length recombinant FVIII
Sponsors
Bayer
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
Inclusion criteria
* Male patients diagnosed with moderate to severe hemophilia A (≤ 5% FVIII:C (Factor VIII Coagulant activity)) * 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 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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Proportion of patients on 2x and 3x weekly prophylaxis at end of observation period | Up to 2 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Annualized composite number of reported bleeds (total, spontaneous, joint and trauma) | Up to 2 years | — |
| Proportion of patients in predefined prophylaxis regimen per age group and per country | At the end of observational period, up to 2 years | Age group: 0 to \<6, ≥6 to \<12, ≥12 to \<18, 18 and above Weekly prophylaxis dosing regimens: * 2 injections a week * 3 injections a week * Injected on every other day |
| Physician decision determinants of prophylaxis regimen | At baseline | Age i.v. access Current treatment regimen Bleeding history with current treatment regimen Prior history of life threatening bleed Number of target joints Pharmacokinetic data Adherence/Compliance history Activity level Patient/caregiver preference Caregiver support Insurance coverage (US) Institution guidelines Country guidelines Other |
| Change from baseline to one year and two years in treatment satisfaction (Hemo-SAT) | At baseline, 1 year and end of observational period, up to 2 years | Hemo-SAT - Hemophilia treatment satisfaction questionnaire |
| Change from baseline to six months, one year and two years in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-PRO) | At baseline, 6 months and end of observational period, up to 2 years | VERITAS - Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis |
| The total annualized factor consumption (injections) | Up to 2 years | — |
| Type of data relating to KOVALTRY PK | At routine visits, up to 2 years | Pharmacokinectic (PK) parameters * Area under the curve (AUC) * Clearance (Cl) * Half-life * FVIII trough * FVIII peak levels * In-vivo recovery |
| Change in prophylaxis dosing frequency (study start to end of observation period) | At baseline and end of observation period, up to 2 years | — |
| Reasons for selection of initial dose / dosing frequency of Kovaltry (study start to end of observation period) | At baseline and end of observation period, up to 2 years | — |
| Number of KOVALTRY PK assessments performed | At routine visits, up to 2 years | — |
| Incidence of adverse events (AEs) and serious adverse events (SAEs) | Up to 2 years | — |
Countries
Belgium, Canada, Colombia, France, Germany, Greece, Italy, Luxembourg, Netherlands, Slovenia, Spain, Taiwan, United States
Outcome results
None listed