Continued Access Study for Patients with Severe Hemophilia A Using Efanesoctocog Alfa

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What is this study about?

This clinical trial is focused on studying a condition known as Hemophilia A, which is a genetic disorder that affects the blood’s ability to clot properly. The treatment being investigated in this study is called efanesoctocog alfa, also known by its code name BIVV001. This medication is designed to help manage bleeding episodes in patients with severe Hemophilia A by providing a replacement for a missing protein that is crucial for blood clotting.

The purpose of this study is to gather more information about the safety and effectiveness of efanesoctocog alfa in patients who have already participated in a previous trial with this treatment. Participants will receive the medication through an intravenous injection, which means it is administered directly into a vein. The study will monitor how well the treatment works in preventing bleeding episodes and how well it is tolerated by the patients over a period of time.

Throughout the study, participants will continue to receive efanesoctocog alfa as a prophylactic treatment, which means it is given regularly to prevent bleeding rather than treating it after it occurs. The study aims to ensure that patients have continued access to this treatment and to collect further data on its long-term use. Participants will be closely monitored for any side effects or adverse events to ensure their safety while receiving the treatment.

1 joining the study

Upon joining the study, the patient must have completed a previous trial with efanesoctocog alfa and be receiving clinical benefits from the treatment.

The patient should enter this study preferably within 7 days of their last dose in the previous study to ensure no interruption in treatment.

2 treatment administration

The patient will receive efanesoctocog alfa, which is a powder for solution for injection.

The medication is administered through an intravenous injection. The frequency and dosage will be determined by the study protocol and the healthcare provider overseeing the trial.

3 monitoring and data collection

Throughout the study, data on the safety and tolerability of efanesoctocog alfa will be collected. This includes monitoring for any adverse events.

The efficacy of the medication as a preventive treatment and in managing bleeding episodes will also be evaluated.

4 patient diary

The patient or their representative will be trained to use a study diary to record relevant information throughout the trial.

This diary is an important tool for tracking the patient’s experience and any symptoms or side effects.

5 completion of the study

The study is estimated to end by March 1, 2027. The patient will continue to receive treatment and monitoring until the study concludes.

Upon completion, the collected data will contribute to understanding the long-term safety and effectiveness of efanesoctocog alfa.

Who Can Join the Study?

  • The patient must be able to give signed informed consent. This means they understand and agree to the study’s rules and requirements. If the patient is under 18 years old or cannot give consent, a parent or legal representative must provide consent. Additionally, if appropriate, the patient under 18 should also agree to participate.
  • Patients must follow local rules about using birth control methods if needed. For males in this study, no birth control measures are required.
  • The patient must have completed one of the required previous studies: Sobi.BIVV001-001, Sobi.BIVV001-003, or LTS16294. They should be benefiting from the treatment with efanesoctocog alfa, as determined by the study doctor. Ideally, the time between the last dose in the previous study and the first visit in this study should be within 7 days for certain studies to avoid stopping the treatment. For another study, they should join this study at the end of the previous study visit.
  • The patient, or their parent or legal representative, must be willing and able to learn how to use the study diary and keep it updated throughout the study.

Who Cannot Join the Study?

  • Patients who have not been previously treated for Haemophilia A. This means they must have received treatment for this condition before.
  • Patients who do not have severe Haemophilia A. This refers to a more serious form of the condition.
  • Patients who are not within the specified age range. The study is for certain age groups only.
  • Patients who are not willing or able to follow the study procedures. This means they must be able to understand and agree to the study requirements.
  • Patients who have other medical conditions that might interfere with the study. This means any other health issues that could affect the study results.
  • Patients who are pregnant or breastfeeding. This is to ensure the safety of both the mother and the baby.
  • Patients who have participated in another clinical trial recently. This is to avoid any interference with the current study.
  • Patients who have a history of allergic reactions to the study medication. This is to prevent any potential allergic response.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Other Sites

Site Name City Country Status
Azienda Unita Locale Socio Sanitaria N 8 Berica Vicenza Italy
Nosokomeio Paidon I Agia Sofia Athens Greece
Region Skane Skanes Universitetssjukhus Lund Sweden
University Multiprofile Hospital For Active Treatment Saint Georgi EAD Plovdiv Bulgaria
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Universitair Medisch Centrum Utrecht Utrecht The Netherlands
Karolinska University Hospital Solna Sweden
Rheinische Friedrich-Wilhelms-Universitaet Bonn Bonn Germany
University Medical Center Ljubljana Ljubljana Slovenia
Centre Hospitalier Lyon Sud Pierre Benite France
Hopital Beaujon Clichy France
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Specialized Hospital For Active Treatment Of Hematological Diseases EAD Sofia Bulgaria
Axyarih Oqqnclavrnu Udultsaiqkurv Pwvit Parma Italy
Lcmpo Gxyyhbv Hpxrkurt Ow Aeimdo Athens Greece
Abjkwompn Ure Amsterdam The Netherlands
Aefhbzveao Pgyxbwwg Hkhkgpck Dz Mpqsyosyt Marseille France
Gwouep Upcxiafzca Fspwhvajb Frankfurt Germany
Useebagski Dkzss Suaio Dh Rjpp Lu Sgwffydc Rome Italy
Ubbszbymfw Mbqqp Gsimjwv Oy Cwurbchtl Catanzaro Italy
Hpbrslsk Uagnmbdqfguux dc A Ccsixg A Coruna Galicia Spain
Hprxqmgt Uotpmjlqlpnmje Seekjvbyti &enctuv Hjyngxl dx Hwhkdsllkfc STRASBOURG, Alsace France

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Bulgaria Bulgaria
Not recruiting
20.03.2024
France France
Not recruiting
20.03.2024
Germany Germany
Not recruiting
20.03.2024
Greece Greece
Not recruiting
20.03.2024
Italy Italy
Recruiting
20.03.2024
Norway Norway
Recruiting
20.03.2024
Slovenia Slovenia
Not recruiting
20.03.2024
Spain Spain
Not recruiting
20.03.2024
Sweden Sweden
Recruiting
20.03.2024
The Netherlands The Netherlands
Not recruiting
20.03.2024

Trial locations

Investigated drugs:

Efanesoctocog alfa is a medication used in the treatment of severe hemophilia A. It is designed to help prevent bleeding episodes and manage bleeding when they occur. This medication works by replacing the missing or deficient factor VIII, a protein that is essential for blood clotting. The trial aims to gather more information on the safety and effectiveness of efanesoctocog alfa in patients who have already been treated with it in previous studies.

Hemophilia A – Hemophilia A is a genetic disorder characterized by a deficiency in clotting factor VIII, which is essential for blood clotting. This condition leads to prolonged bleeding after injuries, surgeries, or even spontaneously without any apparent cause. Individuals with severe hemophilia A may experience frequent bleeding episodes, particularly into joints and muscles, which can cause pain and swelling. Over time, repeated bleeding into joints can lead to chronic joint damage and reduced mobility. The severity of the condition varies, with some individuals experiencing mild symptoms and others having more severe bleeding tendencies. Hemophilia A is typically inherited, affecting mostly males, while females are usually carriers of the gene.

Trial ID:
2023-506537-29-00
Protocol code:
Sobi.BIVV001-002
NCT ID:
NCT06716814
Trial Phase:
Therapeutic confirmatory (Phase III)

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