A Study to Investigate the Course of Synovial Hypertrophy in Patients With Haemophilia A on Efanesoctocog Alfa Prophylaxis

NCT06752850 | Active Not Recruiting Phase 4 FDA Drug

• 2 other identifiers: Sobi.BIVV001-0042024-512066-33-00
• Study Type: interventional
• Target: 37
• Locations: 4 countries
• Sites: 10

Brief Summary

The rationale for conducting this open-label phase 4 study is to assess whether once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) improves the disease course of existing synovial hypertrophy and prevents the risk of joint bleeds in patients with moderate or severe haemophilia A. The use of imaging assessments will allow for objective detection and monitoring of synovial hypertrophy, and thus expand on the previous findings demonstrating positive effects of once-weekly prophylaxis with efanesoctocog alfa (50 IU/kg) on joint health.

Conditions

Haemophilia A (Moderate or Severe); Hemophilia, Classic; Haemophilia; Hemophilia; Classic Hemophilia; Synovial Hypertrophy

Keywords

Deficiency, Factor VIII; Factor 8 Deficiency, Congenital; Factor VIII Deficiency; Factor VIII Deficiency, Congenital; Hemophilia A, Congenital; Synovial Membrane; Joint; Prophylaxis

Outcome Measures

Primary Outcomes (1)

• Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovial hypertrophy domain score decrease.

  At least 1 point decrease in HEAD-US synovial hypertrophy domain score at Month 12 (for joints with a domain score of 1 or 2 at baseline).

  Baseline to 12 months

Secondary Outcomes (12)

• Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovial hypertrophy domain score increase.

  Baseline to 6 or 12 months

• International Prophylaxis Study Group (IPSG) magnetic resonance imaging (MRI)

  Baseline to 12 months

• Haemophilia Joint Health Score (HJHS)

  Baseline to 12 months

• Patient Reported Outcome (PRO) of Pain Intensity

  Baseline to 6 month and 12 months

• Patient Reported Outcome of Pain Interference

  Baseline to 6 month and 12 months

Study Arms (1)

Study Treatment Group

EXPERIMENTAL

Drug: Efanesoctocog alfa

Interventions

Efanesoctocog alfa DRUG

50 IU/kg once weekly, Intravenous injection

Study Treatment Group

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. Parents' or legally designated representatives' consent is required for patients who are \<18 years of age or unable to give consent, or as applicable per local laws. Patients who are \<18 years of age should provide assent in addition to the parents'/legally designated representatives' consent, if appropriate.
• Male or female patients who are ≥12 years of age and diagnosed with moderate or severe haemophilia A (defined as ≤5% of normal FVIII clotting activity) at the time of signing the ICF.
• A female patient is eligible to participate if she is not pregnant at enrolment and does not plan to become pregnant during the study. A woman of child-bearing potential (WOCBP) must have a negative highly sensitive serum pregnancy test at the Screening Visit.
• Must have received prophylactic treatment per local label with any marketed FVIII product or emicizumab for ≥12 months prior to the Baseline Visit.
• Have at least one eligible index joint (ankle, elbow, knee).
• Have 12 months of documented pre-study treatment data on haemophilia prescriptions and on treated bleeding episodes prior to the Baseline Visit.
• Willingness and the ability of the patient or their legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study.

You may not qualify if:

• Blood clotting disorders other than haemophilia A
• Already on efanesoctocog alfa treatment
• Positive inhibitor result (assessed by local laboratory) from the Screening Visit, defined as ≥0.6 Bethesda units (BU)/mL.
• History of inhibitors without successful immune tolerance induction (ITI)
• Successful ITI is defined as:
• Negative inhibitor titer (\<0.6 BU/mL)
• FVIII recovery \> 66% of expected
• FVIII half-life ≥ 6 hours
• ITI performed within the last 2 years prior to the Baseline Visit.
• Currently receiving treatment with any of the prohibited concomitant medications, as specified by the protocol.
• Planned major orthopaedic procedure in any eligible index joint during the course of the study.
• Patients are not eligible for participation in the study if they cannot undergo MRI assessments at the Baseline Visit.
• Patients with known hypersensitivity to the active substance or to any of the excipients.
• 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.

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead
• PSI CRO: collaborator

Study Sites (10)

Sobi Investigational Site

Milan, Italy

Location

Sobi Investigational Site

Naples, Italy

Location

Sobi Investigational Site

Rozzano, Italy

Location

Sobi Investigational Site

Oslo, Norway

Location

Sobi Investigational Site

Barcelona, Spain

Location

Sobi Investigational Site

Madrid, Spain

Location

Sobi Investigational Site

Seville, Spain

Location

Sobi Investigational Site

Valencia, Spain

Location

Sobi Investigational Site

Gothenburg, Sweden

Location

Sobi Investigational Site

Malmö, Sweden

Location

MeSH Terms

Conditions

Hemophilia A; Lymphoma, Follicular; Synovitis

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Joint Diseases; Musculoskeletal Diseases

Study Officials

• Study Physician

  Sobi AB

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 18, 2024
• First Posted: December 31, 2024
• Study Start: December 19, 2024
• Primary Completion (Estimated): November 17, 2026
• Study Completion (Estimated): December 28, 2026
• Last Updated: April 30, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

ES (4); SE (2); NO (1); IT (3)