Clinical trial NCT06716814: Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
Official title: A Multinational, Prospective, Open-label, Roll-over Study (LIBERTY) to Provide Post-trial Access to Treatment for Patients With Severe Haemophilia A Who Have Completed a Previous Trial With Efanesoctocog Alfa
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Severe Haemophilia A
Keywords: Haemophilia A
Blood coagulation disorder
Factor VIII
FVIII
Coagulation protein disorder
Severe Haemophilia A
Efanesoctocog alfa
Lead sponsor: Swedish Orphan Biovitrum
Sponsor class: INDUSTRY
Collaborators: name: PSI CRO
First posted: 2024-12-04
Enrollment: 78
Sex: MALE
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Roll-over study to provide post-trial access to treatment for patients with severe haemophilia A who have completed a previous trial with efanesoctocog alfa.
Primary purpose: TREATMENT

Brief summary: This is a multinational, prospective, open-label, roll-over study in patients with severe 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), or PK comparison study (Sobi.BIVV001-003). 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 Study 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 criteria: 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, or LTS16294, 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 (assessed by central laboratory), defined as ≥0.6 Bethesda units (BU)/mL, at Baseline Visit.
* Ongoing or planned participation in any interventional clinical study at 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.

Primary outcomes:
measure: Number of injections to treat a bleeding episode
timeFrame: From enrollment and up to 52 weeks
measure: Total dose to treat a bleeding episode
timeFrame: From enrollment and up to 52 weeks
measure: Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs)
timeFrame: From enrollment and up to 52 weeks
measure: Annualized bleeding rate (ABR) for treated bleeding episodes
timeFrame: From enrollment and up to 52 weeks
measure: Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed
timeFrame: From enrollment and up to 52 weeks
measure: Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed
timeFrame: From enrollment and up to 52 weeks
measure: Annualized bleeding rate (ABR) for all bleeding episodes
timeFrame: From enrollment and up to 52 weeks
measure: Annualized bleeding rate (ABR) for all bleeding episodes by type of bleed
timeFrame: From enrollment and up to 52 weeks
measure: Annualized bleeding rate (ABR) for all bleeding episodes by location of bleed
timeFrame: From enrollment and up to 52 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Plovdiv
country: Bulgaria
facility: Sobi Investigational Site
city: Sofia
country: Bulgaria
facility: Sobi Investigational Site
city: Bordeaux
country: France
facility: Sobi Investigational Site
city: Brest
country: France
facility: Sobi Investigational Site
city: Bron
country: France
facility: Sobi Investigational Site
city: Le Kremlin-Bicetre
country: France
facility: Sobi Investigational Site
city: Lille
country: France
facility: Sobi Investigational Site
city: Marseille
country: France
facility: Sobi Investigational Site
city: Strasbourg
country: France
facility: Sobi Investigational Site
city: Athens
country: Greece
facility: Sobi Investigational Site
city: Catanzaro
country: Italy
facility: Sobi Investigational Site
city: Florence
country: Italy
facility: Sobi Investigational Site
city: Milano
country: Italy
facility: Sobi Investigational Site
city: Naples
country: Italy
facility: Sobi Investigational Site
city: Parma
country: Italy
facility: Sobi Investigational Site
city: Rome
country: Italy
facility: Sobi Investigational Site
city: Vicenza
country: Italy
facility: Sobi Investigational Site
city: A Coruña
country: Spain
facility: Sobi Investigational Site
city: Valencia
country: Spain
facility: Sobi Investigational Site
city: Zaragoza
country: Spain
facility: Sobi Investigational Site
Study officials:
affiliation: Sobi AB
name: Medical Development Lead
role: STUDY_DIRECTOR
References: