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
- Are there results?
- Not yet: the study is under way
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.
- More trials run by Swedish Orphan BiovitrumThe sponsor's other registered studies.
- 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
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT06716814
- Acronym / sponsor ID
- LIBERTY / Sobi.BIVV001-002
- Conditions
- Haemophilia A (Moderate or Severe)
- Keywords
- Haemophilia A, Blood coagulation disorder, Factor VIII, FVIII, Coagulation protein disorder, Severe Haemophilia A, Efanesoctocog alfa, Haemophilia A (Moderate or Severe)
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 89 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Collaborators
- PSI CRO
- Study start
- 6 Mar 2025 (actual)
- Primary completion
- Mar 2027 (estimated)
- Study completion
- Mar 2027 (estimated)
- First posted
- 4 Dec 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- 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.
| Arm | Type | Receives |
|---|---|---|
| Efanesoctocog alfa prophylaxis | Experimental | Drug: Efanesoctocog alfaPatients 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. |
Who may qualify
- Ages
- 6 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Efanesoctocog alfaAlso: 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. | Drug | Efanesoctocog alfa prophylaxis |
Primary outcome
Number of injections to treat a bleeding episode
Time frame: From enrollment and up to 52 weeks
Total dose to treat a bleeding episode
Time frame: From enrollment and up to 52 weeks
Adverse events (AEs), including serious adverse events (SAEs) and adverse events of special interest (AESIs)
Time frame: From enrollment and up to 52 weeks
Number of participants with occurrence of AEs, SAEs, and AESIs
Annualized bleeding rate (ABR) for treated bleeding episodes
Time frame: From enrollment and up to 52 weeks
ABR for treated bleeding episodes during prophylactic treatment.
Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed
Time frame: From enrollment and up to 52 weeks
ABR for treated bleeding episodes during prophylactic treatment by type of bleed.
Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed
Time frame: From enrollment and up to 52 weeks
ABR for treated bleeding episodes during prophylactic treatment by location of bleed.
Annualized bleeding rate (ABR) for all bleeding episodes
Time frame: From enrollment and up to 52 weeks
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment.
Annualized bleeding rate (ABR) for all bleeding episodes by type of bleed
Time frame: From enrollment and up to 52 weeks
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by type of bleed.
Annualized bleeding rate (ABR) for all bleeding episodes by location of bleed
Time frame: From enrollment and up to 52 weeks
ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment, by location of bleed.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2026.
- Individual participant dataSharing planned: 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
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Development Lead study director
Sobi AB
20 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
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.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in Italy, France, Sweden, Bulgaria, Spain and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of injections to treat a bleeding episode (time frame: From enrollment and up to 52 weeks). The study lists 9 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026; AzTrials last refreshed its copy on 5 Nov 2026.