Clinical and Health-related Outcome of rFVIIIFc Prophylaxis (CLHEAR)

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

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 Overview

• Status: Recruiting
• Conditions: Hemophilia A

• Study Type: Observational
• Enrollment (Anticipated): 48

Contacts and Locations

• Study Contact: Name: Thomas Hilberg, Prof.; Phone Number: 004920237320812; Email: hilberg@uni-wuppertal.de
• Study Contact Backup: Name: Jamil Hmida; Phone Number: 5975 0049202439; Email: jamil.hmida@uni-wuppertal.de

Study Locations

• Germany
  • North Rhine-Westphalia
    • Wuppertal, North Rhine-Westphalia, Germany, 42117
      • Recruiting
      • Department of Sports Medicine, University of Wuppertal
      • Contact: Thomas Hilberg, Prof.; Phone Number: 004920237320812; Email: sportmedizin@uni-wuppertal.de
  • Northwest
    • Wuppertal, Northwest, Germany, 42117
      • Recruiting
      • Department of Sports Medicine
      • Contact: Thomas Hilberg, Prof.; Phone Number: 004920237320812; Email: hilberg@uni-wuppertal.de
      • Contact: Jamil Hmida; Phone Number: 0049202439; Email: jamil.hmida@uni-wuppertal.de

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

• Sampling Method: Non-Probability Sample

Study Population

Patients with moderate or severe Haemophilia A will be included in this study consecutively, who will be switched from FVIII prophylaxis on rFVIIIFc prophylaxis.

Description

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)

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort
Patients with Haemophilia A; Patients suffering from moderate to severe haemophilia A; Age ≥ 18-years; Treatment with FVIII prophylaxis; Submitted written informed consent

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
total annual bleeding rate	number of total bleedings	one year
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
pressure pain thresholds	physiological Parameter in Newton	one year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
spontaneous joint ABR	number of total joint bleedings	one year
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
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
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

Collaborators and Investigators

• Sponsor: Prof. Dr. Dr. Thomas Hilberg
• Investigators: Principal Investigator: Thomas Hilberg, Prof., head of department

Study record dates

Study Major Dates

• Study Start (Actual): February 1, 2021
• Primary Completion (Anticipated): March 31, 2023
• Study Completion (Anticipated): April 20, 2023

Study Registration Dates

• First Submitted: July 28, 2020
• First Submitted That Met QC Criteria: October 6, 2020
• First Posted (Actual): October 12, 2020

Study Record Updates

• Last Update Posted (Actual): May 11, 2021
• Last Update Submitted That Met QC Criteria: May 6, 2021
• Last Verified: May 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A
• Other Study ID Numbers: Hae-Ger-2019-251-Hil

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No