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

Phase 3RecruitingNCT06716814

Swedish Orphan Biovitrum104 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

104

Conditions

Haemophilia A (Moderate or Severe)

Interventions

Eligibility

Sex: MALEMin age: 6 Years

Medical Language ↔ Plain English

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.

Locations (21)

Sobi Investigational Site

Plovdiv, Bulgaria

ACTIVE_NOT_RECRUITING

Sobi Investigational Site

Sofia, Bulgaria

ACTIVE_NOT_RECRUITING

Sobi Investigational Site

Brest, France

ACTIVE_NOT_RECRUITING

Sobi Investigational Site

Bron, France

ACTIVE_NOT_RECRUITING

Sobi Investigational Site

Lille, France

ACTIVE_NOT_RECRUITING

Sobi Investigational Site

Strasbourg, France

WITHDRAWN

Sobi Investigational Site

Athens, Greece

ACTIVE_NOT_RECRUITING

Sobi Investigational Site

Catanzaro, Italy

ACTIVE_NOT_RECRUITING

Sobi Investigational Site

Florence, Italy

ACTIVE_NOT_RECRUITING

Sobi Investigational Site

Milan, Italy

ACTIVE_NOT_RECRUITING

...and 11 more locations