NCT06940830

Long-term Study Evaluating Joint Health in People With Haemophilia A Receiving Real-world Prophylactic Treatment With Efanesoctocog Alfa

A Low-interventional Study to Evaluate Long-term Effectiveness of Real-world Prophylactic Treatment With Efanesoctocog Alfa on Joint Health in People With Haemophilia A (ALTITUDE)

• Status: Recruiting
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 250 (estimated)
• Sponsor: Swedish Orphan Biovitrum · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.

Detailed description

This is a retrospective-prospective, low-interventional study to evaluate the long-term effectiveness of real-world prophylaxis with efanesoctocog alfa on joint health in people with haemophilia A. Participants in the study must be prescribed efanesoctocog alfa at the discretion of their physicians within 6 months prior to, or at the time of, enrolment. The decision to initiate treatment with efanesoctocog alfa shall be made independently from the decision to include the patients in the study. A broad haemophilia A population (e.g. all severities \[mild to severe\], all ages, patients with comorbidities and/or concomitant use of anticoagulant/antiplatelet drugs, patients with previous inhibitors to Factor FVIII \[FVIII\]) will be eligible to enrol. The study will require mandatory on-site visits with joint assessments. A retrospective data collection on patients' haemophilia, medical, and surgical history will be performed, including previous haemophilia treatment and treated bleeding episodes from 12 months prior to initiating treatment with efanesoctocog alfa until enrolment. The study will start with the enrolment visit during a patient's routine visit to the hospital, to check whether the patient fulfils all the inclusion criteria and none of the exclusion criteria. Study participants will have yearly on-site visits for 24 months or 36 months, depending on the time of enrolment in relation to study closure. Patients will be followed in the study as long as they are treated with efanesoctocog alfa prophylaxis which would be at the physicians' discretion. Patients will also need to agree to document any bleeding episodes they may have during the study in a study-specific paper diary. Joint assessment, Haemophilia Joint Health Score (HJHS), and completion of Patient-reported outcomes (PROs) will be conducted during the on-site visits. For patients included in the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) group, ultrasound joint assessment will also be done at the yearly visits. The end of study (EoS) visit can be performed at the 24-month visit or the 36-month visit, depending on the time of enrolment. The target is to enroll approximately 250 participants.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	Efanesoctocog alfa	Participant in this study must be prescribed efanesoctocog alfa prophylactic treatment per their standard of care from their physician. No drug will be provided by the Sponsor.

Timeline

• Start date: 2025-05-21
• Primary completion: 2028-12-01
• Completion: 2028-12-01
• First posted: 2025-04-23
• Last updated: 2026-03-24

Locations

27 sites across 8 countries: Croatia, Czechia, France, Germany, Ireland, Italy, Spain, United Kingdom

Regulatory

• FDA-regulated drug study