Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa

Condition(s) studied

Investigational drug(s) / intervention(s)

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.

Study summary

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.

Eligibility

Primary outcome measure(s)

• Number of injections to treat a bleeding episode — From enrollment and up to 52 weeks
• Total dose to treat a bleeding episode — From enrollment and up to 52 weeks
• Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs) — From enrollment and up to 52 weeks
  Number of participants with occurrence of AEs, SAEs, and AESIs
• Annualized bleeding rate (ABR) for treated bleeding episodes — From enrollment and up to 52 weeks
  ABR for treated bleeding episodes during prophylactic treatment.
• Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed — From enrollment and up to 52 weeks
  ABR for treated bleeding episodes during prophylactic treatment by type of bleed.
• Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed — From enrollment and up to 52 weeks
  ABR for treated bleeding episodes during prophylactic treatment by location of bleed.
• Annualized bleeding rate (ABR) for all bleeding episodes — From enrollment and up to 52 weeks
  ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment.
• Annualized bleeding rate (ABR) for all bleeding episodes by type of bleed — From enrollment and up to 52 weeks
  ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by type of bleed.
• Annualized bleeding rate (ABR) for all bleeding episodes by location of bleed — From enrollment and up to 52 weeks
  ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by location of bleed.

Trial sites (21)

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