MedDataX Logo

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

NCT ID: NCT06940830

Last Updated: 2025-12-17

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

250 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-05-21

Study Completion Date

2028-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

Hemophilia A

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Study Treatment Group

Group Type OTHER

Efanesoctocog alfa

Intervention Type DRUG

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.

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

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.

Intervention Type DRUG

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

PSI CRO

INDUSTRY

Sponsor Role collaborator

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Study Physician

Role: STUDY_DIRECTOR

Swedish Orphan Biovitrum AB (publ)

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Sobi Investigational Site

Zagreb, , Croatia

Site Status RECRUITING

Sobi Investigational Site

Brno, , Czechia

Site Status RECRUITING

Sobi Investigational Site

Ostrava, , Czechia

Site Status RECRUITING

Sobi Investigational Site

Prague, , Czechia

Site Status RECRUITING

Sobi Investigational Site

Bordeaux, , France

Site Status RECRUITING

Sobi Investigational Site

Marseille, , France

Site Status RECRUITING

Sobi Investigational Site

Nantes, , France

Site Status RECRUITING

Sobi Investigational Site

Paris, , France

Site Status NOT_YET_RECRUITING

Sobi Investigational Site

Rennes, , France

Site Status RECRUITING

Sobi Investigational Site

Berlin, , Germany

Site Status RECRUITING

Sobi Investigational Site

Bonn, , Germany

Site Status RECRUITING

Sobi Investigational Site

Frankfurt am Main, , Germany

Site Status RECRUITING

Sobi Investigational Site

Hamburg, , Germany

Site Status RECRUITING

Sobi Investigational Site

Munich, , Germany

Site Status RECRUITING

Sobi Investigational Site

Dublin, , Ireland

Site Status RECRUITING

Sobi Investigational Site

A Coruña, , Spain

Site Status NOT_YET_RECRUITING

Sobi Investigational Site

Barcelona, , Spain

Site Status RECRUITING

Sobi Investigational Site

Málaga, , Spain

Site Status RECRUITING

Sobi Investigational Site

Palma de Mallorca, , Spain

Site Status RECRUITING

Sobi Investigational Site

Zaragoza, , Spain

Site Status RECRUITING

Sobi Investigational Site

Birmingham, , United Kingdom

Site Status RECRUITING

Sobi Investigational Site

Cardiff, , United Kingdom

Site Status RECRUITING

Sobi Investigational Site

London, , United Kingdom

Site Status RECRUITING

Sobi Investigational Site

Manchester, , United Kingdom

Site Status NOT_YET_RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

Croatia Czechia France Germany Ireland Spain United Kingdom

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Study Physician

Role: CONTACT

[email protected]
+46 08-697-20 00

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

2024-517812-31-00

Identifier Type: CTIS

Identifier Source: secondary_id

U1111-1312-9668

Identifier Type: REGISTRY

Identifier Source: secondary_id

Sobi.BIVV001-005

Identifier Type: -

Identifier Source: org_study_id