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Recruiting Phase 3 NCT06716814

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

Phase 3 – large-scale trial before approval
Conditions: Haemophilia A (Moderate or Severe)

Sponsor: Swedish Orphan Biovitrum

trial.available_in: БГ
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.
Who can participate
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.
Interventions
Efanesoctocog alfa
DRUG
Locations 24
Bulgaria (2)
Sobi Investigational Site
Plovdiv
Sobi Investigational Site
Sofia
France (7)
Sobi Investigational Site
Bordeaux
WITHDRAWN
Sobi Investigational Site
Brest
ACTIVE_NOT_RECRUITING
Sobi Investigational Site
Bron
ACTIVE_NOT_RECRUITING
Sobi Investigational Site
Le Kremlin-Bicêtre
WITHDRAWN
Sobi Investigational Site
Lille
ACTIVE_NOT_RECRUITING
Sobi Investigational Site
Marseille
WITHDRAWN
Sobi Investigational Site
Strasbourg
WITHDRAWN
Greece (1)
Sobi Investigational Site
Athens
Italy (8)
Sobi Investigational Site
Catanzaro
Sobi Investigational Site
Florence
Sobi Investigational Site
Milan
Sobi Investigational Site
Naples
Sobi Investigational Site
Parma
Sobi Investigational Site
Rome
Sobi Investigational Site
Rozzano
Sobi Investigational Site
Vicenza
Norway (1)
Sobi Investigational Site
Oslo
Spain (2)
Sobi Investigational Site
A Coruña
ACTIVE_NOT_RECRUITING
Sobi Investigational Site
Zaragoza
ACTIVE_NOT_RECRUITING
Sweden (3)
Sobi Investigational Site
Gothenburg
Sobi Investigational Site
Lund
Sobi Investigational Site
Stockholm
Technical details
Status
Recruiting
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male only
Minimum age
6 Years
Healthy volunteers
No
Start date
06.03.2025
Completion date
01.03.2027
Registry ID
NCT06716814
Source
clinicaltrials.gov
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