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

Completed

• Conditions: Hemophilia A, Congenital
• Interventions: Biological: Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973

• Registration Number: NCT02830477
• Lead Sponsor: Bayer

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.

Study Timeline

• Study First Submitted: 2016-06-29
• Study First Submitted QC: 2016-07-08
• Study First Posted: 2016-07-13
• Study Start: 2016-10-14
• Primary Completion: 2020-12-01
• Study Completion: 2021-03-01
• Status Verified: 2023-11
• Last Update Submitted: 2023-11-03
• Last Update Posted: 2023-11-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: Male
• Target Recruitment: 313

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

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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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
BAY81-8973	Kovaltry (Antihemophilic Factor [Recombinant], BAY81-8973	Previously treated patients receiving IV infusion of KOVALTRY for routine prophylaxis

Primary Outcome Measures

Name	Time	Method
Proportion of patients on 2x and 3x weekly prophylaxis at end of observation period	Up to 2 years

Secondary Outcome Measures

Name	Time	Method
Annualized composite number of reported bleeds (total, spontaneous, joint and trauma)	Up to 2 years
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
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
Incidence of adverse events (AEs) and serious adverse events (SAEs)	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
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
Change in prophylaxis dosing frequency (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

Trial Locations

Locations (13)

East Carolina University - Brody School of Medicine; 🇺🇸 Greenville, North Carolina, United States

Hemophilia Center of Western New York; 🇺🇸 Buffalo, New York, United States

Children's Rehabilitation Services/ University of South Alabama; 🇺🇸 Mobile, Alabama, United States

Nemours Children's Clinic - Division of Pediatric Hematology/Oncology - Jacksonsville; 🇺🇸 Jacksonville, Florida, United States

Nemours Children's Clinic - Pensacola; 🇺🇸 Pensacola, Florida, United States

Comprehensive Center for Bleeding Disorders / Blood Center of Wisconsin; 🇺🇸 Milwaukee, Wisconsin, United States

Henry Ford Hospital Adult Hemophilia and Thrombosis Treatment Center; 🇺🇸 Detroit, Michigan, United States

Children's Hospital at OU Medical Center; 🇺🇸 Oklahoma City, Oklahoma, United States

Intermountain Hemophilia & Thrombosis Center; 🇺🇸 Salt Lake City, Utah, United States

Washington University Center for Bleeding and Blood Clotting Disorders; 🇺🇸 Saint Louis, Missouri, United States

University of Florida Health Cancer Center; 🇺🇸 Gainesville, Florida, United States

Wake Forest University School of Medicine; 🇺🇸 Winston-Salem, North Carolina, United States

University of Colorado Hemophilia and Thrombosis Center; 🇺🇸 Aurora, Colorado, United States