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

Официальное название

A Multinational, Prospective, Open-label, Roll-over Study (LIBERTY) to Provide Post-trial Access to Treatment for Patients With Haemophilia A Who Have Completed a Previous Trial With Efanesoctocog Alfa

Обзор

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.

Вмешательства

• Препарат 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.

Первичные конечные точки

• Number of injections to treat a bleeding episode [Срок оценки: From enrollment and up to 52 weeks]
• Total dose to treat a bleeding episode [Срок оценки: From enrollment and up to 52 weeks]
• Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs) [Срок оценки: From enrollment and up to 52 weeks]
• Annualized bleeding rate (ABR) for treated bleeding episodes [Срок оценки: From enrollment and up to 52 weeks]
• Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed [Срок оценки: From enrollment and up to 52 weeks]
• Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed [Срок оценки: From enrollment and up to 52 weeks]
• Annualized bleeding rate (ABR) for all bleeding episodes [Срок оценки: From enrollment and up to 52 weeks]
• Annualized bleeding rate (ABR) for all bleeding episodes by type of bleed [Срок оценки: From enrollment and up to 52 weeks]
• Annualized bleeding rate (ABR) for all bleeding episodes by location of bleed [Срок оценки: From enrollment and up to 52 weeks]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

Центры проведения

Италия · 8 центров

• 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

Франция · 4 центра

• Sobi Investigational Site — Brest
• Sobi Investigational Site — Bron
• Sobi Investigational Site — Lille
• Sobi Investigational Site — Strasbourg

Швеция · 3 центра

• Sobi Investigational Site — Gothenburg
• Sobi Investigational Site — Lund
• Sobi Investigational Site — Stockholm

Болгария · 2 центра

• Sobi Investigational Site — Plovdiv
• Sobi Investigational Site — Sofia

Испания · 2 центра

• Sobi Investigational Site — A Coruña
• Sobi Investigational Site — Zaragoza

Греция · 1 центр

• Sobi Investigational Site — Athens

Норвегия · 1 центр

• Sobi Investigational Site — Oslo

Идентификаторы

NCT: NCT06716814 · Sobi.BIVV001-002 · 2023-506537-29-00

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

Открыть это исследование на ClinicalTrials.gov ↗