A Phase 4 interventional study of Efanesoctocog alfa in Hemophilia A, sponsored by Swedish Orphan Biovitrum. Active, not recruiting at 31 sites in 8 countries. Per ClinicalTrials.gov, last updated 2026-09-30.
Sponsored by Swedish Orphan Biovitrum · Phase 4, Interventional, and Treatment
Among 11 studies testing efanesoctocog alfa in Hemophilia A — 1 open to participants now, 5 under way, 2 of them phase 3. efanesoctocog alfa · Hemophilia A.
Among the 11 studies testing efanesoctocog alfa in Hemophilia A, this one is the 2nd largest by enrollment, is one of 4 that started in 2025, is one of 3 in phase 4, and was the 10th registered.
844 studies on the registry are indexed under Hemophilia A; 136 are open to participants now.
Browse Hemophilia A studies →1 of the 11 studies testing efanesoctocog alfa in Hemophilia A is open now, in 5 countries. Recruiting first, the newest first:
Sanofi · Sites in United States, Australia, Canada and 2 more countries
Ages 12+
By country, each count listing its studies:
efanesoctocog alfa: first FDA approval 2023 (ALTUVIIIO), first EMA approval 2024. What it is approved for, and how studies give it, is on its page →
Of the 86 Hemophilia A studies recruiting now, 51 (59%) set no upper age limit and 30 (35%) stop at 75 or younger; 54 take children under 18; 10 take healthy volunteers.
This study: all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
11 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 3 · Phase 2 1 · Phase 3 5 · Phase 4 3
First in 2017; this study’s year is marked. 4 started in 2025.
This study's enrollment of 252 is above the median of 30 across 499 interventional studies indexed under Hemophilia A.
Median enrollment 74 across the 11 interventional studies. Of the 6 that completed or were terminated, 3 (50%) have results posted.
This tab as a report to print or save as PDF → With an account.
11 of 11 have an industry lead sponsor. The 3 most frequent:
Swedish Orphan Biovitrum is the lead sponsor of 82 studies on the registry; 6 are open to participants now.
Of its 17 completed or terminated interventional studies of FDA-regulated products, 15 (88%) have results posted.
15 studies in Hemophilia A by Swedish Orphan Biovitrum: none open now, 3 under way, 1 of them phase 3. Of 12 finished, 2 (17%) have results posted.
Among the 15 studies in Hemophilia A by Swedish Orphan Biovitrum, this one is one of 3 that started in 2025, is one of 4 in phase 4, and was the 15th registered.
Swedish Orphan Biovitrum’s Hemophilia A studies by the year they started, since 2014.
4 of the studies testing efanesoctocog alfa in Hemophilia A that are under way reach primary completion within a year, 2 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04644575Phase 3Bioverativ, a Sanofi companyLong-term Safety and Efficacy of Efanesoctocog Alfa (BIVV001) in Previously Treated Patients With Hemophilia A | 2027-01-15 |
| NCT06716814Phase 3Swedish Orphan BiovitrumStudy to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa | 2027-03 |
| NCT06752850Swedish Orphan BiovitrumA Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis | 2026-11-17 |
| NCT06941870SanofiEfanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy | 2027-10-25 |
Add all 4 to your calendar (.ics) → With an account.
Among the 11 studies testing efanesoctocog alfa in Hemophilia A, from their registry updates:
Nothing recorded among them since 2026-09-25.
Among Swedish Orphan Biovitrum’s 15 in Hemophilia A:
Nothing recorded among them since 2026-09-25.
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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.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Data sharing details ↗ · www.sobi.com
No publications or documents are linked to this record.
Started May 2025 (actual). Primary completion is estimated for Dec 2028, 43 months after the start.
Of 39 phase 4 interventional studies under Hemophilia A, half reached primary completion within 31 months of starting (the median), counting those still running; a quarter within 13 and three quarters within 112. This study's planned 43 months is above that.
How these figures are worked out, and which studies are left out of them.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is active, not recruiting, as verified in Sep 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Efanesoctocog alfa