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RECRUITINGPhase 4INTERVENTIONAL

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)

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: 1. Male or female patient with a diagnosis of haemophilia A. 2. Capable of giving signed willing to sign a consent form, which includes compliance with the requirements and restrictions listed in the willing to sign a consent form form (ICF) and in this protocol. 3. Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations. 4. Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment. 5. Have documented pre-study treatment data on haemophilia prophylaxis prescriptions and on treated bleeding episodes from the 12-months prior to being prescribed efanesoctocog alfa treatment and until enrolment into this study. 6. Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs). 7. Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes. 8. For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees). Who Should NOT Join This Trial: 1. Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A. 2. Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit \[BU\]/mL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit. 3. Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study. 4. Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: 1. Male or female patient with a diagnosis of haemophilia A. 2. Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. 3. Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations. 4. Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment. 5. Have documented pre-study treatment data on haemophilia prophylaxis prescriptions and on treated bleeding episodes from the 12-months prior to being prescribed efanesoctocog alfa treatment and until enrolment into this study. 6. Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs). 7. Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes. 8. For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees). Exclusion Criteria: 1. Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A. 2. Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit \[BU\]/mL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit. 3. Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study. 4. Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments.

Treatments Being Tested

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.

Locations (20)

Sobi Investigational Site
Zagreb, Croatia
Sobi Investigational Site
Brno, Czechia
Sobi Investigational Site
Ostrava, Czechia
Sobi Investigational Site
Prague, Czechia
Sobi Investigational Site
Bordeaux, France
Sobi Investigational Site
Marseille, France
Sobi Investigational Site
Montpellier, France
Sobi Investigational Site
Nantes, France
Sobi Investigational Site
Paris, France
Sobi Investigational Site
Rennes, France
Sobi Investigational Site
Rouen, France
Sobi Investigational Site
Berlin, Germany
Sobi Investigational Site
Bonn, Germany
Sobi Investigational Site
Frankfurt am Main, Germany
Sobi Investigational Site
Hamburg, Germany
Sobi Investigational Site
Munich, Germany
Sobi Investigational Site
Dublin, Ireland
Sobi Investigational Site
Turin, Italy
Sobi Investigational Site
A Coruña, Spain
Sobi Investigational Site
Barcelona, Spain