Brief Title
Clinical and Health-related Outcome of rFVIIIFc Prophylaxis
Official Title
Clinical and Health-related Outcome of rFVIIIFc Prophylaxis in Patients With Haemophilia A
Brief Summary
Current standard therapy for patients with haemophilia (PwH) in the prevention of bleeding episodes is a prophylactic intravenous treatment with recombinant coagulation factor (F) VIII (Haemophilia A) or rather FIX (Haemophilia B) two to three times weekly. With the development of recombinant factor VIII Fc fusion protein (rFVIIIFc) the conventional routine prophylaxis regime is complemented by an extended half-life (EHL) factor replacement prophylaxis with the potential of improved bleed prevention and reduced injection frequency at similar factor consumption. Aim of this longitudinal multicentre study is to evaluate the influence of an EHL factor replacement regime with rFVIIIFc on haemophilic specific parameters (annual bleeding rate, bleeding localisation), joint status, pain, functional parameters, treatment adherence and health-related quality of life in PwH A.
Study Type
Observational
Primary Outcome
total annual bleeding rate
Secondary Outcome
spontaneous joint ABR
Condition
Hemophilia A
Study Arms / Comparison Groups
Patients with Haemophilia A
Description: Patients suffering from moderate to severe haemophilia A Age ≥ 18-years Treatment with FVIII prophylaxis Submitted written informed consent
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
48
Start Date
February 1, 2021
Completion Date
April 20, 2023
Primary Completion Date
March 31, 2023
Eligibility Criteria
Inclusion Criteria: - Patients suffering from moderate to severe haemophilia A - Age ≥ 18-years - Treatment with FVIII prophylaxis - Submitted written informed consent Exclusion Criteria: Patients suffering from other bleeding diseases - Patients with inhibitors - Patients without written informed consent - Age < 18-year-old - Any surgeries up to 6 months before the examination date - Suffering from different rheumatologic diseases like M. Bechterew, Psoriasis or other local or generalized joint infections (Borreliosis, septic arthritis)
Gender
Male
Ages
18 Years - N/A
Accepts Healthy Volunteers
No
Contacts
Thomas Hilberg, Prof., 004920237320812, [email protected]
Location Countries
Germany
Location Countries
Germany
Administrative Informations
NCT ID
NCT04583930
Organization ID
Hae-Ger-2019-251-Hil
Responsible Party
Sponsor-Investigator
Study Sponsor
Prof. Dr. Dr. Thomas Hilberg
Study Sponsor
Thomas Hilberg, Prof., Principal Investigator, Head of Department
Verification Date
May 2021