Haemophilia A (Moderate or Severe)Phase 3LIBERTYNCT06716814
Study to Provide Continued Access to Treatment for Patients Completing a Previous Trial With Efanesoctocog Alfa
A Phase 3 study testing Efanesoctocog alfa in people with Haemophilia A (Moderate or Severe), run by Swedish Orphan Biovitrum at 20 sites in 7 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 years and older, men only
- Conditions the study is for
- Haemophilia A (Moderate or Severe)
- What is being tested?
- Efanesoctocog alfa
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 89 participants
- Where
- 20 sitesin 7 countries
- Study dates
- 6 Mar 2025to Mar 2027
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
20 sites in 7 countries.
20 sites in 7 countries
Italy8 sites
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
France3 sites
Sobi Investigational Site
Brest
Sobi Investigational Site
Bron
Sobi Investigational Site
Lille
Sweden3 sites
Sobi Investigational Site
Gothenburg
Sobi Investigational Site
Lund
Sobi Investigational Site
Stockholm
Bulgaria2 sites
Sobi Investigational Site
Plovdiv
Sobi Investigational Site
Sofia
Spain2 sites
Sobi Investigational Site
A Coruña
Sobi Investigational Site
Zaragoza
About this study
Official title: 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
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.
- Conditions
- Haemophilia A (Moderate or Severe)
- Being tested
- Efanesoctocog alfa
- Keywords
- Haemophilia A, Blood coagulation disorder, Factor VIII, FVIII, Coagulation protein disorder, Severe Haemophilia A, Efanesoctocog alfa, Haemophilia A (Moderate or Severe)
- Medical subject headings
- Hemophilia A, Lymphoma, Follicular, Blood Coagulation Disorders, Coagulation Protein Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Efanesoctocog alfaDrug
Also called BIVV001, Altuvoct, Altuviiio, Recombinant coagulation factor VIII Fc-von-Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN)
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.
| Study group | Role | What they receive |
|---|---|---|
| Efanesoctocog alfa prophylaxis | Experimental | Patients 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. Receives: Drug: Efanesoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of injections to treat a bleeding episode
Measured: From enrollment and up to 52 weeks
Total dose to treat a bleeding episode
Measured: 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
Measured: From enrollment and up to 52 weeks
Annualized bleeding rate (ABR) for treated bleeding episodes
ABR for treated bleeding episodes during prophylactic treatment.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: From enrollment and up to 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Dec 2024
- Study start6 Mar 2025
- Primary completionMar 2027 (estimated)
- Study completionMar 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia A (Moderate or Severe)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- Roll-over study to provide post-trial access to treatment for patients with haemophilia A who have completed a previous trial with efanesoctocog alfa.
- Masking
- None (open label)
- Enrolment
- 89 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PSI CRO
- Responsible party
- Sponsor
- Investigators
- Medical Development Lead, Study director, Sobi AB
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06716814
- Sponsor's protocol ID
- Sobi.BIVV001-002
- Other IDs
- 2023-506537-29-00, 2023
- Acronym
- LIBERTY
- Status last verified
- Oct 2026
- First submitted
- 2 Oct 2024
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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