Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis

Bayer313 enrolled

Overview

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.

Open label, prospective, non-interventional, single arm study in patients receiving KOVALTRY as prophylaxis therapy.

Study Type

OBSERVATIONAL

Enrollment

313

Conditions

Hemophilia A, Congenital

Interventions

Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973BIOLOGICAL

unmodified, full length recombinant FVIII

Eligibility

Sex: MALE

Medical Language ↔ Plain English

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

Locations (25)

Children's Rehabilitation Services/ University of South Alabama

Mobile, Alabama, United States

Outcomes

Primary Outcomes

Proportion of patients on 2x and 3x weekly prophylaxis at end of observation period

Time frame: Up to 2 years

Secondary Outcomes

Annualized composite number of reported bleeds (total, spontaneous, joint and trauma)

Time frame: Up to 2 years

Proportion of patients in predefined prophylaxis regimen per age group and per country

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

Time frame: At the end of observational period, up to 2 years

Physician decision determinants of prophylaxis regimen

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

Time frame: At baseline

Change from baseline to one year and two years in treatment satisfaction (Hemo-SAT)

Hemo-SAT - Hemophilia treatment satisfaction questionnaire

Time frame: At baseline, 1 year and end of observational period, up to 2 years

Change from baseline to six months, one year and two years in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-PRO)

VERITAS - Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis

Time frame: At baseline, 6 months and end of observational period, up to 2 years

Incidence of adverse events (AEs) and serious adverse events (SAEs)

Data from ClinicalTrials.gov

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