Clinical trial NCT02830477: Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis
Official title: A Multinational Phase IV Study Evaluating "Real World" Treatment Pattern in Previously Treated Hemophilia A Patients Receiving KOVALTRY (Octocog Alfa) for Routine Prophylaxis
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A, Congenital
Keywords: Previously treated patients
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2016-07-13
Enrollment: 313
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: OTHER
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Proportion of patients on 2x and 3x weekly prophylaxis at end of observation period
timeFrame: Up to 2 years
Secondary outcomes:
measure: Annualized composite number of reported bleeds (total, spontaneous, joint and trauma)
timeFrame: Up to 2 years
measure: Proportion of patients in predefined prophylaxis regimen per age group and per country
timeFrame: At the end of observational period, up to 2 years
measure: Physician decision determinants of prophylaxis regimen
timeFrame: At baseline
measure: Change from baseline to one year and two years in treatment satisfaction (Hemo-SAT)
timeFrame: At baseline, 1 year and end of observational period, up to 2 years
measure: Change from baseline to six months, one year and two years in Validated Hemophilia Regimen Treatment Adherence Scale-Prophylaxis (VERITAS-PRO)
timeFrame: At baseline, 6 months and end of observational period, up to 2 years
measure: Incidence of adverse events (AEs) and serious adverse events (SAEs)
timeFrame: Up to 2 years
measure: Type of data relating to KOVALTRY PK
timeFrame: At routine visits, up to 2 years
measure: The total annualized factor consumption (injections)
timeFrame: Up to 2 years
measure: Change in prophylaxis dosing frequency (study start to end of observation period)
timeFrame: At baseline and end of observation period, up to 2 years
measure: Reasons for selection of initial dose / dosing frequency of Kovaltry (study start to end of observation period)
timeFrame: At baseline and end of observation period, up to 2 years
measure: Number of KOVALTRY PK assessments performed
timeFrame: At routine visits, up to 2 years
Other outcomes:
Locations:
city: Mobile
country: United States
facility: Children's Rehabilitation Services/ University of South Alabama
state: Alabama
city: Aurora
country: United States
facility: University of Colorado Hemophilia and Thrombosis Center
state: Colorado
city: Gainesville
country: United States
facility: University of Florida Health Cancer Center
state: Florida
city: Jacksonville
country: United States
facility: Nemours Children's Clinic - Division of Pediatric Hematology/Oncology - Jacksonsville
state: Florida
city: Pensacola
country: United States
facility: Nemours Children's Clinic - Pensacola
state: Florida
city: Detroit
country: United States
facility: Henry Ford Hospital Adult Hemophilia and Thrombosis Treatment Center
state: Michigan
city: Saint Louis
country: United States
facility: Washington University Center for Bleeding and Blood Clotting Disorders
state: Missouri
city: Buffalo
country: United States
facility: Hemophilia Center of Western New York
state: New York
city: Greenville
country: United States
facility: East Carolina University - Brody School of Medicine
state: North Carolina
city: Winston-Salem
country: United States
facility: Wake Forest University School of Medicine
state: North Carolina
city: Oklahoma City
country: United States
facility: Children's Hospital at OU Medical Center
state: Oklahoma
city: Salt Lake City
country: United States
facility: Intermountain Hemophilia & Thrombosis Center
state: Utah
city: Milwaukee
country: United States
facility: Comprehensive Center for Bleeding Disorders / Blood Center of Wisconsin
state: Wisconsin
city: Multiple Locations
country: Belgium
city: Multiple Locations
country: Canada
city: Multiple Locations
country: Colombia
city: Multiple Locations
country: France
city: Multiple Locations
country: Germany
city: Multiple Locations
country: Greece
city: Multiple Locations
country: Italy
city: Multiple Locations
country: Luxembourg
city: Multiple Locations
country: Netherlands
city: Multiple Locations
country: Slovenia
city: Multiple Locations
country: Spain
city: Multiple Locations
country: Taiwan
Study officials:
affiliation: Bayer
name: Bayer Study Director
role: STUDY_DIRECTOR
References:
citation: Santoro C, Fuh B, Le PQ, Maes P, Berrueco R, Mingot-Castellano EM, von Mackensen S, Tueckmantel C, Cabre-Marquez JF, Wang M. Efficacy and safety in patients with haemophilia A switching to octocog alfa (BAY 81-8973): Final results of the global real-world study, TAURUS. Eur J Haematol. 2023 Jan;110(1):77-87. doi: 10.1111/ejh.13876. Epub 2022 Oct 17.