Clinical and Health-related Outcome of rFVIIIFc Prophylaxis

Hemophilia A Clinical Trial

— CLHEAR  

Official title:

Clinical and Health-related Outcome of rFVIIIFc Prophylaxis in Patients With Haemophilia A

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04583930
• Other study ID #: Hae-Ger-2019-251-Hil
• Status: Recruiting
• Start date: February 1, 2021
• Est. completion date: April 20, 2023

Study information

• Verified date: May 2021
• Source: University of Wuppertal
• Contact: Thomas Hilberg, Prof.
• Phone: 004920237320812
• Email: hilberg[@]uni-wuppertal.de
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 48
• Est. completion date: April 20, 2023
• Est. primary completion date: March 31, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

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)

Related Conditions & MeSH terms

• Hemophilia A

Locations

Country	Name	City	State
Germany	Department of Sports Medicine	Wuppertal	Northwest
Germany	Department of Sports Medicine, University of Wuppertal	Wuppertal	North Rhine-Westphalia

Sponsors (1)

Lead Sponsor	Collaborator
Prof. Dr. Dr. Thomas Hilberg

Country where clinical trial is conducted

Germany, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	total annual bleeding rate	number of total bleedings	one year
Primary	orthopaedic joint status (Haemophilia health joint score)	The clinical joint status will be examined in all patients with haemophilia by the World Federation Joint Examination Score and Haemophilia Joint Health Score. Higher score points imply an increased deficit in the functional and structural joint status as a sign of more pronounced haemophilic arthropathy, with a maximum possible value of 124 (no deficits = 0).	one year
Primary	pressure pain thresholds	physiological Parameter in Newton	one year
Secondary	spontaneous joint ABR	number of total joint bleedings	one year
Secondary	subjective quality of life (SF-36)	The standarized questionnaire SF-36 includes a physical and psychological score. The higher the score, the higher the physical or psychological quality of life. A higher score implies a better physical and mental quality of life	two years
Secondary	subjective physical performance (HEP-Test-Q)	The final version of the standarized questionnaire consists of 25 items pertaining to the domains 'mobility', 'strength & coordination', 'endurance' and 'body perception'. The response options were a five-point Likert scale (ranging from 1 = never to 5 = always). Some of the items had to be re-coded; subscales and the total score were transformed to a scale of 0-100 with high scores indicating better physical performance	two years
Secondary	self-perceived functional abilities (Haemophilia Activities List)	The Haemophilia Activities List measures the impact of hemophilia on self-perceived functional abilities in adults. It contains 42 multiple choice questions in seven domains: Lying/sitting/kneeling/standing (8 items), Functions of the legs (9 items), Functions of the arms (4 items), Use of transportation (3 items), Self-care (5 items), Household tasks (6 items), Leisure activities and sports (7 items)	two years