NCT05856266

An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa

An 18-month Low-interventional Prospective, Multicentre Study to Assess Joint Outcomes in Patients With Haemophilia A or B on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa

• Status: Terminated
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 10 (actual)
• Sponsor: Swedish Orphan Biovitrum · Industry
• Sex: All
• Age: 6 Years
• Healthy volunteers: Not accepted

Summary

The goal of this low-interventional study is to describe the overall joint health in patients with haemophilia A or haemophilia B prophylactically treated with rFVIIIFc or rFIXFc. The main question it aims to answer is the: • Evaluation of the overall joint status as detected by ultrasound in haemophilia A and B patients treated with rFVIIIFc or rFIXFc prophylaxis over the 18-month study period. Participants will come to 6-monthly visits during the 18-month long study period and will perform an ultrasound with the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) protocol at each visit. At baseline and end of study visits, the patients will be assessed with the clinical scoring system Haemophilia Joint Health Score (HJHS) and complete patient questionnaires. Retrospective data from patient medical records will also be collected for at least 6 months before enrolment in the study.

Detailed description

The main purpose of this study is to prospectively describe the joint health over an 18-month period of prophylactic treatment with rFVIIIFc or rFIXFc in patients with haemophilia A or haemophilia B in a real-world setting in Europe. This is a low-interventional, multicentre study using point-of-care US examination for regular monitoring of joint health and detection of hypertrophic synovium, cartilage and bone damage. At the same time, clinical joint status will also be examined by HJHS. This study will evaluate the presence, resolution, recurrence, and new development of target joints. It will also describe bleeding episodes and evaluate the quality of life and physical activity with PROs. If patients are using the CE marked Florio HAEMO app in their routine clinical practice, they will be offered to participate in an optional sub-study that aims to explore possible correlations between the levels of physical activity, the estimated FVIII/FIX levels and bleeding occurrence.

Conditions

• Hemophilia A
• Hemophilia B

Interventions

Type	Name	Description
PROCEDURE	Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)	Ultrasound examination
OTHER	Haemophilia Joint Health Score (HJHS)	Functional joint examination

Timeline

• Start date: 2023-08-24
• Primary completion: 2023-12-06
• Completion: 2023-12-06
• First posted: 2023-05-12
• Last updated: 2025-01-16
• Results posted: 2025-01-16

Locations

56 sites across 11 countries: Bulgaria, Croatia, Czechia, France, Hungary, Ireland, Italy, Romania, Slovenia, Spain, United Kingdom