NCT06579144

Brief 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. 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.

Trial Health

80
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
24

participants targeted

Target at P25-P50 for phase_1

Timeline
0mo left

Started Feb 2025

Geographic Reach
4 countries

8 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress96%
Feb 2025May 2026

First Submitted

Initial submission to the registry

August 28, 2024

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 30, 2024

Completed
6 months until next milestone

Study Start

First participant enrolled

February 24, 2025

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 30, 2026

Completed
28 days until next milestone

Study Completion

Last participant's last visit for all outcomes

May 28, 2026

Expected
Last Updated

February 18, 2026

Status Verified

December 1, 2025

Enrollment Period

1.2 years

First QC Date

August 28, 2024

Last Update Submit

February 13, 2026

Conditions

Hemophilia A

Keywords

Haemophilia ABlood coagulation disorderefanesoctocog alfaFVIIICoagulation protein disorderPharmacokinetics

Outcome Measures

Primary Outcomes (1)

  • 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 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.

    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.

    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.

    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.

    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.

    up to 7 days and 14 days after the administration of the respective drugs.

  • +4 more secondary outcomes

Study Arms (2)

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.

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.

Drug: Efanesoctocog alfa

Interventions

Efanesoctocog alfaDRUG

Recombinant coagulation factor VIII Fc-von Willebrand Factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN)

Also known as: BIVV001, Altuvoct, Altuviiio
Damactocog alfa pegolTuroctocog alfa pegol

Eligibility Criteria

Age18 Years - 65 Years
Sexmale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (8)

Sobi Investigational Site

Sofia, Bulgaria

RECRUITING

Sobi Investigational site

Frankfurt, Germany

RECRUITING

Sobi Investigational Site

Oldenburg, Germany

RECRUITING

Sobi Investigational Site

Milan, Italy

RECRUITING

Sobi Investigational Site

Naples, Italy

NOT YET RECRUITING

Sobi Investigational Site

A Coruña, Spain

RECRUITING

Sobi Investigational Site

Valencia, Spain

RECRUITING

Sobi Investigational Site

Zaragoza, Spain

RECRUITING

MeSH Terms

Conditions

Hemophilia ABlood Coagulation DisordersCoagulation Protein Disorders

Interventions

BIVV001

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedHematologic DiseasesHemic and Lymphatic DiseasesHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Elena Santagostino, MD

    Sobi AB

    STUDY DIRECTOR

Central Study Contacts

Blank Clinical Study Physician, MD PhD

CONTACT

+46 8 697 20 00medical.info@sobi.com

Clinical Program Leader

CONTACT

+46 8 697 20 00medical.info@sobi.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
OTHER
Intervention Model
SEQUENTIAL
Model Details: Open-label, 2-period, fixed-sequence study for intra-participant comparison of PK-profiles.
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 28, 2024

First Posted

August 30, 2024

Study Start

February 24, 2025

Primary Completion

April 30, 2026

Study Completion (Estimated)

May 28, 2026

Last Updated

February 18, 2026

Record last verified: 2025-12

Data Sharing

IPD Sharing
Will share

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

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
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
More information

Locations

IT(2)BU(1)DE(2)ES(3)