Skip to content
Clinical Trials – home

Rare diseases

Investigational molecules

Locations

Our offerNewsroom

RecruitingRare disease

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

Fast replyRegistered drug
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

What is this trial about?

A plain-language summary of the goals, design and what participants do

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.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 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. Step 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. Step 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. Step 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. Step 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 trial?

4 criteria

  • 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 trial?

8 criteria

  • 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.
Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

We usually reply within a few days

Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Trial locations

Where you can join this trial

Countries are shaded by recruitment status. Click a recruiting country to ask about joining there.

RecruitingNot recruiting
Not finding your country?

Not sure what to do next?

Joining a clinical trial can seem overwhelming. We guide you step by step, so you know exactly what to expect and how we support you through the process.

See the full process and FAQ

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.

What is already known about the treatment

Efanesoctocog alfa – This medication is administered through intravenous infusion and is currently being studied in clinical trials for its safety and effectiveness. It is primarily used for patients with severe Hemophilia A, a condition that affects blood clotting. Efanesoctocog alfa works by replacing the missing or deficient factor VIII, a protein essential for blood clotting, thereby helping to prevent and control bleeding episodes. It is classified as a recombinant factor VIII therapy, which is a type of medication used to treat bleeding disorders.

Investigated diseases

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 detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-506537-29-00Protocol codeSobi.BIVV001-002Estimated enrolment131 patientsSponsorSwedish Orphan Biovitrum AB (publ)

sourced from the EU Clinical Trials Register and site verification

Want to learn more about this trial or check if you can participate?

Clinical Trials Concierge

Prefer not to search? Our Concierge searches the trials for you.

Tell us about your condition – we search every trial in Europe and connect you with the right site.

ClinicalTrials.eu

Every clinical trial in Europe, in plain language.

Explore the full ecosystem of clinical research in Europe.

Search trials

Legal notice · Published by CTIN POLAND sp. z o.o., ul. rtm. Witolda Pileckiego 67/109, 02-781 Warsaw, Poland · KRS 0001111334 · REGON 528919042 · NIP 9512598637

© 2026 Clinical Trials EU – European Clinical Trials Information Network

GDPR compliance, ISO 9001 and ISO 27001 certified (LL-C Certification)

On this site, “treatment” means an investigational medicine being studied in a clinical trial. Its safety and efficacy for the use being studied have not yet been confirmed, some participants may receive a placebo or a comparator medicine, and taking part does not guarantee any health benefit. The decision to take part is made by the doctor at the research site. This site is for information only and does not replace medical advice.

This service is not affiliated with the European Commission, the EMA, or the official CTIS system. Most information comes from publicly available international clinical-trial registries, supplemented by data from academic sites, national regulators and commercial sponsors. On this site, “treatment” and “therapy” mean a medicine being tested in a clinical trial. Its safety and effectiveness in the use being studied are not yet confirmed, some participants may receive a placebo or a comparator, and taking part does not guarantee a health benefit. The doctor at the research site decides who can take part. This site provides information, not medical advice. Certain content and visual elements on this website have been generated or enhanced using artificial intelligence (AI).