Hemophilia APhase 1NCT06579144
Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A
A Phase 1 study testing Efanesoctocog alfa in people with Hemophilia A, run by Swedish Orphan Biovitrum at 7 sites in 4 countries.
Trial summary
Sobi.BIVV001-003 is an open-label, 2-period, fixed sequence study for intra-participant comparison of the PK profiles of efanesoctocog alfa and the extended half-life rFVIII products damactocog alfa pegol or turoctocog alfa pegol after a single i.v. Read more about this study
- Who is it for?
- Ages 18 to 65 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Efanesoctocog alfa
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 7 sitesin 4 countries
- Study dates
- 24 Feb 2025to 21 May 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 18 to 65 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant must be male, 18 to 65 years of age, inclusive, at the time of signing the informed consent form (ICF).
- Severe haemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII activity, as documented in historical medical records from a clinical laboratory demonstrating <1% FVIII coagulant activity or a documented genotype known to produce severe haemophilia A.
- Previous treatment for haemophilia A with any marketed recombinant and/or plasma derived FVIII for at least 150 exposure days.
- Currently receiving treatment with damoctocog alfa pegol or turoctocog alfa pegol at Screening.
Who cannot join
- Any history of a positive inhibitor test, defined as >0.6 Bethesda units (BU)/mL in at least two consecutive Bethesda inhibitor assays, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL. Family history of inhibitors will not exclude the participant.
- Positive FVIII inhibitor result (assessed by central laboratory), defined as ≥0.6 BU/mL at Screening.
Trial locations and contacts
7 sites in 4 countries.
7 sites in 4 countries
Spain3 sites
Sobi Investigational Site
A Coruña
Sobi Investigational Site
Valencia
Sobi Investigational Site
Zaragoza
Germany2 sites
Sobi Investigational Site
Bonn
Sobi Investigational site
Frankfurt
About this study
Official title: An Open-label, Multicentre Study to Compare the Pharmacokinetics of Efanesoctocog Alfa Versus rFVIII Products, Damactocog Alfa Pegol or Turoctocog Alfa Pegol, After a Single Intravenous Dose of 50 IU/kg in a Fixed Sequence in Previously Treated Adults With Severe Haemophilia A.
Sobi.BIVV001-003 is an open-label, 2-period, fixed sequence study for intra-participant comparison of the PK profiles of efanesoctocog alfa and the extended half-life rFVIII products damactocog alfa pegol or turoctocog alfa pegol after a single i.v. injection in previously treated males, 18-65 years of age, with severe haemophilia A.
Participants who are receiving treatment with damoctocog alfa pegol (n~12) or turoctocog alfa pegol (n~12) will be enrolled in the study. The study will start with a screening period (up to 28 days), including a wash-out period prior to start of the actual study period.
Read more
During the the first visit, a single dose of damactocog alfa pegol or turoctocog alfa pegol (corresponding to the participant's pre-study treatment) will be administered. A PK sampling period will follow over 7 visits. Following completion of the PK sampling of the original treatment regimen, the patients will be given a single dose of efanesoctocog alfa at visit 8, after which a new PK sampling period will follow (visit 8-15).
The primary objective for the study is to compare the half-life of efanesoctocog alfa with that of the two comparator drugs after a single iv. injections.
Secondary objectives include comparison of area under the curve for efanesoctocog alfa vs. the two comparator drugs, characterization of PK parameters for all three drugs as well as well as to evaluate safety and tolerability of a single iv. injection of efanesoctocog alfa.
- Conditions
- Hemophilia A
- Being tested
- Efanesoctocog alfa
- Keywords
- Haemophilia A, Blood coagulation disorder, efanesoctocog alfa, FVIII, Coagulation protein disorder, Pharmacokinetics
- Medical subject headings
- Hemophilia A, 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)
| Study group | Role | What they receive |
|---|---|---|
| Damactocog alfa pegol | Active comparator | Patients treated with damactocog alfa pegol at the time of screening will receive one single injection with 50 IU/kg at visit 1, then one single dose 50 IU/kg with efanesoctocog alfa at visit 8. Receives: Drug: Efanesoctocog alfa |
| Turoctocog alfa pegol | Active comparator | Patients treated with turoctocog alfa pegol at the time of screening will receive one single injection with 50 IU/kg at visit 1, then one single dose 50 IU/kg with efanesoctocog alfa at visit 8. Receives: Drug: Efanesoctocog alfa |
What is being measured
The primary outcome is the main question the study is designed to answer.
Half-life (t½) of efanesoctocog alfa, damactocog alfa pegol and turoctocog alfa pegol after a single i.v. injection
PK assessments will be based on FVIII activity levels determined by one-stage clotting assay
Measured: up to 7 days and 14 days after the administration of the respective drugs.
Secondary outcomes (9)
To compare the area under the curve zero to infinity (AUC∞) of efanesoctocog alfa with that of damoctocog alfa pegol and with that of turoctocog alfa pegol after a single i.v. injection.
The area under the curve will be calculated based on drug concentration analyses from time zero to last quantifiable sample collection timepoint
Measured: up to 7 days and 14 days after the administration of the respective drugs.
To characterize Cmax of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Maximum concentration observed (Cmax) assessed by FVIII activity measurement by one-stage aPTT clotting assay
Measured: up to 7 days and 14 days after the administration of the respective drugs.
To characterize clearance of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Clearance of drug/s (CL) assessed by FVIII activity measurement by one-stage aPTT clotting assay
Measured: up to 7 days and 14 days after the administration of the respective drugs.
To characterize volume of distribution of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Volume of distribution (Vd) assessed by FVIII activity measurement by one-stage aPTT clotting assay
Measured: up to 7 days and 14 days after the administration of the respective drugs.
To characterize mean residence time of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Mean residence time (MRT) assessed by FVIII activity measurement by one-stage aPTT clotting assay
Measured: up to 7 days and 14 days after the administration of the respective drugs.
To characterize incremental recovery of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Incremental Recovery (IR) assessed by FVIII activity measurement by one-stage aPTT clotting assay
Measured: up to 7 days and 14 days after the administration of the respective drugs.
To characterize time to specific plasma FVIII levels of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Time to specific plasma FVIII:C levels (40%, 20%, and 10%) assessed by FVIII activity measurement by one-stage aPTT clotting assay
Measured: up to 7 days and 14 days after the administration of the respective drugs.
To characterize time spent in plasma FVIII:C normal range for efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Time to spent in plasma FVIII:C normal range (50% - 150%) assessed by FVIII activity measurement by one-stage aPTT clotting assay
Measured: up to 7 days and 14 days after the administration of the respective drugs.
To evaluate the safety and tolerability of a single IV injection of efanesoctocog alfa.
All safety data from visit 1 up until the dose of efanesoctocog alfa for the first treatment period and then subsequently all safety data from the dose of efanesoctocog alfa until EoS will be analyzed for this outcome measure.
Measured: From first dose of comparator to end of study, approximately 1 month.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Aug 2024
- Study start24 Feb 2025
- Primary completion21 May 2026
- Study completion21 May 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies 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 1
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Open-label, 2-period, fixed-sequence study for intra-participant comparison of PK-profiles.
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Swedish Orphan Biovitrum
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PSI CRO
- Responsible party
- Sponsor
- Investigators
- Elena Santagostino, MD, Study director, Sobi AB
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06579144
- Sponsor's protocol ID
- Sobi.BIVV001-003
- Status last verified
- Jun 2026
- First submitted
- 28 Aug 2024
- Last updated
- 25 Jun 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's 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 25 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-label, Multicentre Study to Compare the Pharmacokinetics of Efanesoctocog Alfa Versus rFVIII Products, Damactocog Alfa Pegol or Turoctocog Alfa Pegol, After a Single Intravenous Dose of 50 IU/kg in a Fixed Sequence in Previously Treated Adults With Severe Haemophilia A.
- ClinicalTrials.gov ID
- NCT06579144
- Acronym / sponsor ID
- Sobi.BIVV001-003
- Conditions
- Hemophilia A
- Keywords
- Haemophilia A, Blood coagulation disorder, efanesoctocog alfa, FVIII, Coagulation protein disorder, Pharmacokinetics
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- Swedish Orphan BiovitrumIndustry (pharma, biotech, devices)
- Collaborators
- PSI CRO
- Study start
- 24 Feb 2025 (actual)
- Primary completion
- 21 May 2026 (actual)
- Study completion
- 21 May 2026 (actual)
- First posted
- 30 Aug 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.
Sobi.BIVV001-003 is an open-label, 2-period, fixed sequence study for intra-participant comparison of the PK profiles of efanesoctocog alfa and the extended half-life rFVIII products damactocog alfa pegol or turoctocog alfa pegol after a single i.v. injection in previously treated males, 18-65 years of age, with severe haemophilia A.
Participants who are receiving treatment with damoctocog alfa pegol (n~12) or turoctocog alfa pegol (n~12) will be enrolled in the study. The study will start with a screening period (up to 28 days), including a wash-out period prior to start of the actual study period.
During the the first visit, a single dose of damactocog alfa pegol or turoctocog alfa pegol (corresponding to the participant's pre-study treatment) will be administered. A PK sampling period will follow over 7 visits. Following completion of the PK sampling of the original treatment regimen, the patients will be given a single dose of efanesoctocog alfa at visit 8, after which a new PK sampling period will follow (visit 8-15).
The primary objective for the study is to compare the half-life of efanesoctocog alfa with that of the two comparator drugs after a single iv. injections.
Secondary objectives include comparison of area under the curve for efanesoctocog alfa vs. the two comparator drugs, characterization of PK parameters for all three drugs as well as well as to evaluate safety and tolerability of a single iv. injection of efanesoctocog alfa.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Open-label, 2-period, fixed-sequence study for intra-participant comparison of PK-profiles.
| Arm | Type | Receives |
|---|---|---|
| Damactocog alfa pegol | Active comparator | Drug: Efanesoctocog alfaPatients treated with damactocog alfa pegol at the time of screening will receive one single injection with 50 IU/kg at visit 1, then one single dose 50 IU/kg with efanesoctocog alfa at visit 8. |
| Turoctocog alfa pegol | Active comparator | Drug: Efanesoctocog alfaPatients treated with turoctocog alfa pegol at the time of screening will receive one single injection with 50 IU/kg at visit 1, then one single dose 50 IU/kg with efanesoctocog alfa at visit 8. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Participant must be male, 18 to 65 years of age, inclusive, at the time of signing the informed consent form (ICF).
- Severe haemophilia A, defined as <1 IU/dL (<1%) endogenous FVIII activity, as documented in historical medical records from a clinical laboratory demonstrating <1% FVIII coagulant activity or a documented genotype known to produce severe haemophilia A.
- Previous treatment for haemophilia A with any marketed recombinant and/or plasma derived FVIII for at least 150 exposure days.
- Currently receiving treatment with damoctocog alfa pegol or turoctocog alfa pegol at Screening.
Key exclusion criteria (2 listed)
- Any history of a positive inhibitor test, defined as >0.6 Bethesda units (BU)/mL in at least two consecutive Bethesda inhibitor assays, or any value greater than or equal to the lower sensitivity cut-off for laboratories with cut-offs for inhibitor detection between 0.7 and 1.0 BU/mL. Family history of inhibitors will not exclude the participant.
- Positive FVIII inhibitor result (assessed by central laboratory), defined as ≥0.6 BU/mL at Screening.
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, AltuviiioRecombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN) | Drug | Damactocog alfa pegol, Turoctocog alfa pegol |
Primary outcome
Half-life (t½) of efanesoctocog alfa, damactocog alfa pegol and turoctocog alfa pegol after a single i.v. injection
Time frame: up to 7 days and 14 days after the administration of the respective drugs.
PK assessments will be based on FVIII activity levels determined by one-stage clotting assay
9 secondary outcomes
To compare the area under the curve zero to infinity (AUC∞) of efanesoctocog alfa with that of damoctocog alfa pegol and with that of turoctocog alfa pegol after a single i.v. injection.
Time frame: up to 7 days and 14 days after the administration of the respective drugs.
To characterize Cmax of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Time frame: up to 7 days and 14 days after the administration of the respective drugs.
To characterize clearance of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Time frame: up to 7 days and 14 days after the administration of the respective drugs.
To characterize volume of distribution of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Time frame: up to 7 days and 14 days after the administration of the respective drugs.
To characterize mean residence time of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Time frame: up to 7 days and 14 days after the administration of the respective drugs.
To characterize incremental recovery of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Time frame: up to 7 days and 14 days after the administration of the respective drugs.
To characterize time to specific plasma FVIII levels of efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Time frame: up to 7 days and 14 days after the administration of the respective drugs.
To characterize time spent in plasma FVIII:C normal range for efanesoctocog alfa and damoctocog alfa pegol or turoctocog alfa pegol after a single IV injection.
Time frame: up to 7 days and 14 days after the administration of the respective drugs.
To evaluate the safety and tolerability of a single IV injection of efanesoctocog alfa.
Time frame: From first dose of comparator to end of study, approximately 1 month.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Jun 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's 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.
Elena Santagostino, MD study director
Sobi AB
7 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Efanesoctocog alfa in people with Hemophilia A, run by Swedish Orphan Biovitrum at 7 sites in 4 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 4 inclusion and 2 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: finished, as of the sponsor's last update on 25 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Spain, Germany, Bulgaria, Italy. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Half-life (t½) of efanesoctocog alfa, damactocog alfa pegol and turoctocog alfa pegol after a single i.v. injection (time frame: up to 7 days and 14 days after the administration of the respective drugs.). 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 25 Jun 2026.