A Phase 3 interventional study of Efanesoctocog alfa in Haemophilia A (Moderate or Severe), sponsored by Swedish Orphan Biovitrum. Recruiting at 21 sites in 7 countries. Open to male participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-06-26.
Sponsored by Swedish Orphan Biovitrum · Phase 3, Interventional, and Treatment
This is a multinational, prospective, open-label, roll-over study in patients with haemophilia A, ≥6 years of age, who have completed participation in any of the parental studies with efanesoctocog alfa; XTEND-ed study (LTS16294), FREEDOM study (Sobi.BIVV001-001), PK comparison study (Sobi.BIVV001-003) or SHINE study (Sobi.BIVV001-004). The aim of the study is to provide patients with continuous benefit from efanesoctocog alfa treatment and to further continue clinical monitoring for safety and efficacy until efanesoctocog alfa is commercially available in each patient's respective country (or until March 2027, whichever comes first).
The study starts with the Baseline Visit, which will be done in connection to the end of treatment at the EoT/EoS visit (or equivalent) in the respective parent study. Subsequent study visits (on site or phone call) will be done approximately every 13 weeks until End of Treatment. An End of Study safety phone call will be done 14 (+7) days after the End of Treatment Visit.
Exclusion Criteria:
Patients who have completed a previous study with efanesoctocog alfa may continue to be treated with a prophylactic dose of 50 IU/kg efanesoctocog alfa once weekly.
Drug: Efanesoctocog alfa
Treatment starts when the patient completes the parent study and continues until efanesoctocog alfa is commercially available in each patient's respective country, or until March 2027, whichever comes first.
Also known as: BIVV001, Altuvoct, Altuviiio, Recombinant coagulation factor VIII Fc-von-Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN)
Number of injections to treat a bleeding episode
Time frame: From enrollment and up to 52 weeks
Total dose to treat a bleeding episode
Time frame: From enrollment and up to 52 weeks
Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs)
Number of participants with occurrence of AEs, SAEs, and AESIs
Time frame: From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes
ABR for treated bleeding episodes during prophylactic treatment.
Time frame: From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed
ABR for treated bleeding episodes during prophylactic treatment by type of bleed.
Time frame: From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed
ABR for treated bleeding episodes during prophylactic treatment by location of bleed.
Time frame: From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for all bleeding episodes
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment.
Time frame: From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for all bleeding episodes by type of bleed
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by type of bleed.
Time frame: From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for all bleeding episodes by location of bleed
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by location of bleed.
Time frame: From enrollment and up to 52 weeks
Plan to share: Yes — Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sobi data sharing criteria and process for requesting access can be found at: https:// www.sobi.com/en/policies
Supporting information: Study protocol, Sap, Icf, Csr
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Swedish Orphan Biovitrum