Pharmacokinetic Comparison of Efanesoctocog Alfa vs Other EHL-rFVIII Products in Participants With Severe Haemophilia A

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.

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 Overview

• Status: Recruiting
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: Efanesoctocog alfa

• Study Type: Interventional
• Enrollment (Estimated): 24
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Blank Clinical Study Physician, MD PhD; Phone Number: +46 8 697 20 00; Email: medical.info@sobi.com
• Study Contact Backup: Name: Clinical Program Leader; Phone Number: +46 8 697 20 00; Email: medical.info@sobi.com

Study Locations

• Bulgaria
  • Sofia, Bulgaria
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
• Germany
  • Bonn, Germany
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Frankfurt, Germany
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
• Italy
  • Milan, Italy
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Naples, Italy
    • Not yet recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
• Spain
  • A Coruña, Spain
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Valencia, Spain
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator
  • Zaragoza, Spain
    • Recruiting
    • Sobi Investigational Site
    • Contact: Principal Investigator

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Damactocog alfa pegol; 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.	Drug: Efanesoctocog alfa; Recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN); Other Names: BIVV001; Altuviiio; Altuvoct
Active Comparator: Turoctocog alfa pegol; 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.	Drug: Efanesoctocog alfa; Recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN); Other Names: BIVV001; Altuviiio; Altuvoct

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	up to 7 days and 14 days after the administration of the respective drugs.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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	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	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	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	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	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	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	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	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.	From first dose of comparator to end of study, approximately 1 month.

Collaborators and Investigators

• Sponsor: Swedish Orphan Biovitrum
• Collaborators: PSI CRO
• Investigators: Study Director: Elena Santagostino, MD, Sobi AB

Study record dates

Study Major Dates

• Study Start (Actual): February 24, 2025
• Primary Completion (Estimated): May 30, 2026
• Study Completion (Estimated): May 30, 2026

Study Registration Dates

• First Submitted: August 28, 2024
• First Submitted That Met QC Criteria: August 28, 2024
• First Posted (Actual): August 30, 2024

Study Record Updates

• Last Update Posted (Actual): May 14, 2026
• Last Update Submitted That Met QC Criteria: May 12, 2026
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Keywords: Pharmacokinetics; FVIII; Haemophilia A; Blood coagulation disorder; Coagulation protein disorder; efanesoctocog alfa
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia A; Blood Coagulation Disorders; Coagulation Protein Disorders; BIVV001
• Other Study ID Numbers: Sobi.BIVV001-003

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: 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
• IPD Sharing Time Frame: Within 1 year following completion of the trial, or at the time of CSR finalization.

IPD Sharing 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

• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: Yes