Haemophilia A (Moderate or Severe)
Conditions
Keywords
Haemophilia A, Blood coagulation disorder, Factor VIII, FVIII, Coagulation protein disorder, Severe Haemophilia A, Efanesoctocog alfa, Haemophilia A (Moderate or Severe)
Brief 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.
Interventions
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.
Sponsors
Swedish Orphan Biovitrum
PSI CRO
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Roll-over study to provide post-trial access to treatment for patients with haemophilia A who have completed a previous trial with efanesoctocog alfa.
Eligibility
Sex/Gender
MALE
Age
6 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Capable of giving signed informed consent. Parents or legally designated representatives' consent is required for patients who are below 18 years of age or unable to give consent. Patients who are below 18 years of age may provide assent in addition to the parents'/legally designated representatives' consent, if appropriate. * Must have completed one of the required parent studies: Sobi.BIVV001-001, Sobi.BIVV001-003, LTS16294, or Sobi.BIVV001-004, and be receiving a clinical benefit from the efanesoctocog alfa treatment, as judged by the Investigator. * Willingness and ability of patient or their parent or legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study.
Exclusion criteria
* Positive inhibitor result, defined as ≥0.6 Bethesda units (BU)/mL, present at the Baseline Visit. * Ongoing or planned participation in any interventional clinical study at the Baseline Visit. * Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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. |
Countries
Bulgaria, France, Greece, Italy, Norway, Spain, Sweden
Contacts
CONTACTStudy Physician
CONTACTClinical Program Lead
STUDY_DIRECTORMedical Development Lead
Sobi AB
Outcome results
None listed