Study on Long-Term Effects of Efanesoctocog Alfa in Preventing Joint Bleeds in Patients with Hemophilia A

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What is this study about?

This clinical trial focuses on studying the long-term effects of a treatment called efanesoctocog alfa in people with hemophilia A. Hemophilia A is a genetic disorder where blood does not clot properly, leading to excessive bleeding. The treatment being tested is a type of medication known as a coagulation factor VIII, which helps the blood to clot and is given as an injection into a vein.

The purpose of this study is to evaluate how well efanesoctocog alfa works in preventing joint bleeds over a long period. Participants in the study will receive regular injections of the medication and will be monitored for any bleeding episodes, particularly in their joints. The study will also look at how the treatment affects joint health over time, including any changes in joint condition and the number of bleeding events.

Participants will be observed for several years to gather information on the effectiveness of the treatment. The study will track the number of bleeding episodes, the amount of medication needed to control bleeding, and any changes in joint health. This information will help to understand the benefits of using efanesoctocog alfa as a regular preventive treatment for people with hemophilia A.

1 enrollment

Upon joining the study, the patient will be enrolled after confirming a diagnosis of haemophilia A and providing informed consent. The patient must have been prescribed efanesoctocog alfa for prophylactic treatment within the last six months or at the time of enrollment.

The patient must have a history of receiving prophylactic treatment for at least 12 months prior to being prescribed efanesoctocog alfa and must provide documented data on previous treatments and bleeding episodes.

2 treatment administration

The patient will receive efanesoctocog alfa as a solution for injection. The medication is administered through an intravenous injection. The specific dosage will be determined based on the patient’s needs and the product available, which includes options like 250 IU, 500 IU, 1,000 IU, 2,000 IU, 3,000 IU, and 4,000 IU.

The frequency and duration of the treatment will be in accordance with the study protocol and the patient’s individual treatment plan.

3 regular assessments

The patient will undergo regular assessments to monitor joint health and the effectiveness of the treatment. This includes the Haemophilia Joint Health Score (HJHS) and patient-reported outcomes (PROs).

For those in the HEAD-US subgroup, an annual ultrasound assessment of the index joints (ankles, elbows, knees) will be conducted.

4 documentation

The patient or their legally designated representative is required to document all bleeding episodes, including any treatments for those episodes. This documentation is crucial for evaluating the treatment’s effectiveness.

5 follow-up visits

The patient will attend follow-up visits as per the study schedule. These visits are essential for ongoing monitoring and assessment of the patient’s condition and response to treatment.

Changes in the Haemophilia Joint Health Score (HJHS) will be evaluated at months 12, 24, and 36 to assess joint health over time.

6 end of study

The study is estimated to conclude by December 2, 2028. At the end of the study, the patient’s overall health and treatment outcomes will be reviewed to determine the long-term effectiveness of efanesoctocog alfa in preventing joint bleeds.

Who Can Join the Study?

Male or female patient with a diagnosis of Haemophilia A.
Capable of giving signed informed consent, which means you understand the study and agree to follow its rules.
Prescribed with efanesoctocog alfa (a type of medication) for regular treatment within 6 months before joining the study or at the latest during the first visit, following local rules.
Must have been on regular treatment with any haemophilia product for at least 12 months before starting efanesoctocog alfa treatment.
Have records of treatment and bleeding episodes from the 12 months before starting efanesoctocog alfa treatment until joining the study.
Willing to follow the visit schedule and undergo required study assessments, such as HJHS (a joint health score) and PROs (patient-reported outcomes).
Willing and able, or have a representative who is willing and able, to document all bleeding episodes and any treatments for those episodes.
For those in the HEAD-US subgroup: Agree to have an annual ultrasound check on specific joints (ankles, elbows, knees).

Who Cannot Join the Study?

Patients who do not have Haemophilia A cannot participate. Haemophilia A is a condition where blood does not clot properly, leading to excessive bleeding.
Patients who are not within the specified age range cannot participate. The study includes specific age groups, so if you are outside these age ranges, you cannot join.
Patients who are not willing to follow the study procedures and requirements cannot participate. This means you must be ready to attend all study visits and follow instructions given by the study team.
Patients who have other medical conditions that might interfere with the study cannot participate. This means if you have other health issues that could affect the study results, you may not be eligible.
Patients who are currently participating in another clinical trial cannot participate. If you are already part of another study, you cannot join this one at the same time.
Patients who have had a recent major surgery or are planning to have surgery during the study period cannot participate. Major surgery can affect your health and the study results.
Patients who are pregnant or breastfeeding cannot participate. This is to ensure the safety of both the mother and the baby.
Patients who have a history of allergic reactions to the study medication cannot participate. If you have had allergies to similar medications, you may not be eligible.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy
CHU de Rouen – Hôpital Charles Nicolle	Rouen	France
Hospital Universitario De Salamanca	Salamanca	Spain
Centre Hospitalier Universitaire De Bordeaux	Bordeaux	France

Other Sites

Site Name	City	Country
Azienda Ospedaliero-Universitaria Policlinico G. Rodolico-San Marco Di Catania	Catania	Italy
Azienda Ospedaliera Universitaria San Giovanni Di Dio E Ruggi d’Aragona	Salerno	Italy
Azienda Ospedaliera di Padova	Padua	Italy
KBC Zagreb	Zagreb	Croatia
Charite Universitaetsmedizin Berlin KöR	Berlin	Germany
Fakultni Nemocnice Brno	Brno	Czechia
Karolinska University Hospital	Solna	Sweden
Centre Hospitalier Universitaire De Montpellier	Montpellier	France
Rheinische Friedrich-Wilhelms-Universitaet Bonn	Bonn	Germany
Centre Hospitalier Universitaire De Nantes	Nantes	France
Centre Hospitalier Universitaire De Rennes	Rennes	France
Hopital Beaujon	Clichy	France
Servei De Salut De Les Illes Balears	Palma	Spain
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen	Gothenburg	Sweden
University Hospital Ostrava	Ostrava	Czechia
Children’s Health Ireland	Dublin	Ireland
HZRM Haemophilie-Zentrum Rhein Main GmbH	Mörfelden-Walldorf	Germany
Udapzrbjmw Mhabqhc Ctxrdk Haipzbabsmymrzbpk	Hamburg	Germany
Finoayds nfrwurlac Msucd a Hfnekbg	Prague	Czechia
Auyvpsvxbi Pbjacjrp Hjopsunk Du Mcvgdnths	Marseille	France
Anakajx Ovussmlczkn Ufhgvsyqlojwo Clgmbbdcikaz Dxcxz Svqznb E Dmxrh Stvnoal De Tbrhwv	Turin	Italy
Kmudpmld dbd Uvjdacbugybd Mtnwuhph Adk	Munich	Germany
Aourwew Ufvfe Sdgxhzaei Lxport Dn Blisacw	Bologna	Italy
Hhgphjlr Dr Lv Ssabd Ckzw I Skga Pxv	Barcelona	Spain
Hhulljho Uxaynwefboicd dw A Cpgixa	A Coruna Galicia	Spain

Trial status

Country	Status	Recruitment Start
Croatia	Recruiting	22.05.2025
Czechia	Recruiting	22.05.2025
France	Recruiting	22.05.2025
Germany	Recruiting	22.05.2025
Ireland	Recruiting	22.05.2025
Italy	Recruiting	22.05.2025
Spain	Recruiting	22.05.2025
Sweden	Not recruiting	22.05.2025

Trial locations

Investigated drugs:

Efanesoctocog Alfa

Efanesoctocog alfa is a medication used in this clinical trial to help people with Hemophilia A. Hemophilia A is a condition where the blood does not clot properly, leading to excessive bleeding, especially in the joints. This medication is given to patients regularly to prevent joint bleeds. It works by replacing a missing protein in the blood that helps with clotting, which can help reduce the number of bleeding episodes and improve joint health over time.

Haemophilia A – Haemophilia A is a genetic disorder caused by a deficiency of clotting factor VIII, which is essential for blood clotting. This condition leads to prolonged bleeding episodes, especially after injuries or surgeries. Individuals with Haemophilia A may experience spontaneous bleeding into joints, muscles, and other tissues, which can cause pain and swelling. Over time, repeated joint bleeds can lead to joint damage and reduced mobility. The severity of the disease varies, with some individuals experiencing frequent bleeding episodes, while others have milder symptoms. It is an inherited condition, typically passed from parents to children through genes.

Trial ID:

2024-517812-31-00

Protocol code:

Sobi.BIVV001-005

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Long-Term Effects of Efanesoctocog Alfa in Preventing Joint Bleeds in Patients with Hemophilia A

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