ClinicalTrials.gg
Active, not recruitingNCT06716814LIBERTYUpdated Oct 19, 2026

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

A Phase 3 interventional study of Efanesoctocog alfa in Haemophilia A (Moderate or Severe), sponsored by Swedish Orphan Biovitrum. Active, not recruiting at 20 sites in 7 countries. Open to male participants aged 6 Years and older. Per ClinicalTrials.gov, last updated 2026-10-19.

Sponsored by Swedish Orphan Biovitrum · Phase 3, Interventional, and Treatment

Updated Oct 19, 2026Now Active, not recruitingEnrollment updated+1 moreGo to Updates ↓
Phase
Phase 3
Study type
Interventional
Enrollment
89
Allocation
Not applicable
Ages
6 Years and older
Sex
Male
01

Study 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.

02

Conditions studied

  • 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)
03

In context

Hemophilia A

844 studies on the registry are indexed under Hemophilia A; 138 are open to participants now.

This study's enrollment of 89 is above the median of 30 across 499 interventional studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Swedish Orphan Biovitrum is the lead sponsor of 82 studies on the registry; 6 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 15 (88%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Years and older
Sexes eligible
Male
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Capable of giving signed . 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

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.
Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
89 participants (actual)

Study arms

  • Experimental
    Efanesoctocog alfa prophylaxis

    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.

    Drug: Efanesoctocog alfa

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.

    Also known as: BIVV001, Altuvoct, Altuviiio, Recombinant coagulation factor VIII Fc-von-Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN)

06

What researchers measure

Primary outcomes

  1. Number of injections to treat a bleeding episode

    Time frame: From enrollment and up to 52 weeks

  2. Total dose to treat a bleeding episode

    Time frame: From enrollment and up to 52 weeks

  3. 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

    Time frame: From enrollment and up to 52 weeks

  4. Annualized bleeding rate (ABR) for treated bleeding episodes

    ABR for treated bleeding episodes during prophylactic treatment.

    Time frame: From enrollment and up to 52 weeks

  5. Annualized bleeding rate (ABR) for treated bleeding episodes by type of bleed

    ABR for treated bleeding episodes during prophylactic treatment by type of bleed.

    Time frame: From enrollment and up to 52 weeks

  6. Annualized bleeding rate (ABR) for treated bleeding episodes by location of bleed

    ABR for treated bleeding episodes during prophylactic treatment by location of bleed.

    Time frame: From enrollment and up to 52 weeks

  7. Annualized bleeding rate (ABR) for all bleeding episodes

    ABR for all bleeding episodes (including untreated bleeding episodes, excluding surgery bleeds) during prophylactic treatment.

    Time frame: From enrollment and up to 52 weeks

  8. 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.

    Time frame: From enrollment and up to 52 weeks

  9. 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.

    Time frame: From enrollment and up to 52 weeks

07

Study locations

20 sites
  • Sobi Investigational Site
    Plovdiv, Bulgaria
  • Sobi Investigational Site
    Sofia, Bulgaria
  • Sobi Investigational Site
    Brest, France
  • Sobi Investigational Site
    Bron, France
  • Sobi Investigational Site
    Lille, 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
08

References and documents

Individual participant data

Plan to share: 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

Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report

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.

Data sharing details ↗ · www.sobi.com

No publications or documents are linked to this record.

09

Updates

1 registry update since Sep 25, 2026
Status
Recruiting→Active, not recruiting
changed Oct 19, 2026
Sites
1 site removed
Show 1 removed
  • Sobi Investigational Site · Strasbourg, France
Oct 19, 2026
Enrollment
104 (estimated)→89 (actual)
Oct 19, 2026
Show all 1 update
  1. Oct 19, 2026
    Recruiting→Active, not recruiting
    1 site removed
    Show 1 removed
    • Sobi Investigational Site · Strasbourg, France
    Enrollment 104 (estimated)→89 (actual)
    + 3 other changes: identifiers, verification date and contact details

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT06716814
Collaborators
PSI CRO
Responsible party
Sponsor
First posted
Dec 4, 2024
Start date
Mar 6, 2025
Primary completion
Mar 2027 (estimated)
Completion
Mar 2027 (estimated)
Last update
Oct 19, 2026

Study contacts

Medical Development Lead
study director · Sobi AB

Oversight

Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.

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Discussion

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Studies of the drug tested here: Efanesoctocog alfa

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