A Phase 4 interventional study of Efanesoctocog alfa in Hemophilia A, sponsored by Swedish Orphan Biovitrum. Recruiting at 29 sites in 8 countries. Per ClinicalTrials.gov, last updated 2026-06-30.
Sponsored by Swedish Orphan Biovitrum · Phase 4, Interventional, and Treatment
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.
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.
Exclusion Criteria:
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.
Annualized joint bleeding rate (AjBR) over the prospective observation period.
Time frame: Enrolment to Month 12, 24 and 36
Annualized bleeding rate (ABR) over the prospective observation period.
Time frame: Enrolment to Month 12, 24 and 36
Number of injections to control a bleeding event over the prospective observation period.
Time frame: Enrolment to Month 12, 24 and 36
Total dose to control bleeding event over the prospective observation period.
Cumulative amount of medication administered to effectively manage bleeding episodes.
Time frame: Enrolment to Month 12, 24 and 36
Haemophilia Joint Health Score (HJHS) version 2.1
Change from enrolment in total and domain scores of HJHS per patient.
Time frame: Enrolment to Month 12, 24 and 36
Target joint development on a patient and joint level over the prospective observation period.
A target joint is defined as joints with 3 or more spontaneous bleeding episodes within a consecutive 6-month period.
Time frame: Month 12, 24 and 36
Target joint resolution on a patient and joint level over the prospective observation period.
A target joint is defined as joints with 3 or more spontaneous bleeding episodes within a consecutive 6-month period.
Time frame: Month 12, 24 and 36
Target joint recurrency on a patient and joint level over the prospective observation period.
A target joint is defined as joints with 3 or more spontaneous bleeding episodes within a consecutive 6-month period.
Time frame: Month 12, 24 and 36
Annualised injection frequency over the prospective observation period.
Time frame: Month 12, 24 and 36
Annualised efanesoctocog alfa consumption for prophylaxis and treatment of bleeds (excluding surgery) over the prospective observation period.
Time frame: Month 12, 24 and 36
Reason(s) for any changes in prescribed frequency and/or dose of efanesoctocog alfa prophylaxis over the prospective observation period.
Time frame: Enrolment to Month 12, 24 and 36
Change in ABR in the period of using efanesoctocog alfa vs prior haemophilia treatment.
Time frame: Prospective period vs Retrospective period
Change in annualised injection frequency during the period on efanesoctocog alfa vs the period on prior haemophilia treatment.
Time frame: Prospective period vs Retrospective period
Change in annualised consumption during the period on efanesoctocog alfa vs the period on prior haemophilia treatment.
Time frame: Prospective period vs Retrospective period
Reason(s) for Switching to efanesoctocog alfa treatment.
Time frame: Retrospective period (12 to 18 months prior to enrollment)
Investigator or Surgeon assessment of patient's haemostatic response on the International Society on Thrombosis and Haemostasis (ISTH) 4-point response for surgical procedures scale (Intraoperative and postoperative).
Time frame: Within 96 hours following surgery
Number of injections and dose administered to maintain haemostasis during perioperative period for minor and major surgery.
Time frame: Enrolment to 36 Months
Total efanesoctocog alfa consumption during perioperative period for minor/major surgery.
Time frame: Enrolment to 36 Months
Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)
Change from enrolment in total and domain score.
Time frame: Enrolment to Month 12, 24 and 36
Treatment preference survey
Time frame: Month 12
Patient Reported Outcome (PRO) of Pain Intensity
Use of PROMIS SF v2.0 to measure change in patient-reported pain intensity from enrolment.
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome (PRO) of Pain Interference
Use of PROMIS SF v2.0 to measure change in patient-reported pain interference from enrolment.
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of 5-level EuroQol-5 dimensions [EQ-5D-5L] score
Changes from enrolment of patient mobility, self-care, usual activities, pain/discomfort, anxiety/depression.
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of youth EuroQol 5-dimensions [EQ-5D-Y] score
Changes from enrolment of patient mobility, self-care, usual activities, pain/discomfort, anxiety/depression using child-appropriate wording.
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of Parent Proxy EuroQol 5-dimensions [EQ-5D-Y:1] score
Parent or caregiver's rating of the child's health outcomes based on mobility, self-care, usual activities, pain/discomfort, anxiety/depression.
Time frame: Enrolment to Month 12, 24 and 36
Patient Reported Outcome of ACTIVLIM-Hemo.
Measurement of activity limitations for patients with haemophilia (PwH).
Time frame: Enrolment to Month 12, 24 and 36
Number of participants with occurrence of Serious Adverse Events (SAEs).
Time frame: Enrolment to 36 months
Number of participants with occurrence of Adverse Events of Special Interest (AESIs).
Time frame: Enrolment to 36 months
Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents. Patient level data will be anonymized and study documents, if applicable will be redacted to protect the privacy of trial participants.
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Swedish Orphan Biovitrum