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RECRUITINGPhase 3INTERVENTIONAL

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

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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - Capable of giving signed willing to sign a consent form. 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. Who Should NOT Join This Trial: - 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
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.

Treatments Being Tested

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.

Locations (20)

Sobi Investigational Site
Plovdiv, Bulgaria
Sobi Investigational Site
Sofia, Bulgaria
Sobi Investigational Site
Bordeaux, France
Sobi Investigational Site
Brest, France
Sobi Investigational Site
Bron, France
Sobi Investigational Site
Le Kremlin-Bicêtre, France
Sobi Investigational Site
Lille, France
Sobi Investigational Site
Marseille, France
Sobi Investigational Site
Strasbourg, France
Sobi Investigational Site
Athens, Greece
Sobi Investigational Site
Catanzaro, Italy
Sobi Investigational Site
Florence, Italy
Sobi Investigational Site
Milan, Italy
Sobi Investigational Site
Naples, Italy
Sobi Investigational Site
Parma, Italy
Sobi Investigational Site
Rome, Italy
Sobi Investigational Site
Rozzano, Italy
Sobi Investigational Site
Vicenza, Italy
Sobi Investigational Site
Oslo, Norway
Sobi Investigational Site
A Coruña, Spain