Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
LIBERTY
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
2 other identifiers
interventional
104
7 countries
24
Brief Summary
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Mar 2025
24 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
October 2, 2024
CompletedFirst Posted
Study publicly available on registry
December 4, 2024
CompletedStudy Start
First participant enrolled
March 6, 2025
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
March 1, 2027
April 20, 2026
September 1, 2025
2 years
October 2, 2024
April 15, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (9)
Number of injections to treat a bleeding episode
From enrollment and up to 52 weeks
Total dose to treat a bleeding episode
From enrollment and up to 52 weeks
Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs)
Number of participants with occurrence of AEs, SAEs, and AESIs
From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes
ABR for treated bleeding episodes during prophylactic treatment.
From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed
ABR for treated bleeding episodes during prophylactic treatment by type of bleed.
From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed
ABR for treated bleeding episodes during prophylactic treatment by location of bleed.
From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for all bleeding episodes
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment.
From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for all bleeding episodes by type of bleed
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by type of bleed.
From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for all bleeding episodes by location of bleed
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by location of bleed.
From enrollment and up to 52 weeks
Study Arms (1)
Efanesoctocog alfa prophylaxis
EXPERIMENTALPatients who have completed a previous study with efanesoctocog alfa may continue to be treated with a prophylactic dose of 50 IU/kg efanesoctocog alfa once weekly.
Interventions
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.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Swedish Orphan Biovitrumlead
- PSI CROcollaborator
Study Sites (24)
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
Sobi Investigational Site
Zaragoza, Spain
Sobi Investigational Site
Gothenburg, Sweden
Sobi Investigational Site
Lund, Sweden
Sobi Investigational Site
Stockholm, Sweden
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Development Lead
Sobi AB
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
October 2, 2024
First Posted
December 4, 2024
Study Start
March 6, 2025
Primary Completion (Estimated)
March 1, 2027
Study Completion (Estimated)
March 1, 2027
Last Updated
April 20, 2026
Record last verified: 2025-09
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Time Frame
- Within 1 year following completion of the trial, or at the time of CSR finalization.
- Access Criteria
- A decision on sharing will be based on the following: The scientific merit of the proposal - i.e. the proposal should be scientifically sound, ethical, and have the potential to contribute to the advancement of public health. The feasibility of the research proposal - i.e. the requesting research team must be scientifically qualified and have the resources to conduct the proposed project. Maintenance of personal integrity - i.e. Sobi will not consider sharing individual data if there is a risk of re-identification of individuals despite a proper anonymisation. Moreover, the patients' informed consent will always be respected. Sobi reserves the right to reject the proposal if the anonymisation process will render unusable data. Publication of results - the applicants should commit to submit their findings to a peer-reviewed scientific journal, alternatively to present the results at a congress (poster or similar), regardless of the research outcome.
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sobi data sharing criteria and process for requesting access can be found at: https:// www.sobi.com/en/policies