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NCT Number: NCT05911763

A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A

This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan.

Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Sex eligibility

All sexes

Study type

Observational

Primary location

Banner MD Anderson Cancer Center- Site Number : 8400008, Gilbert, Arizona, United States

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Have a diagnosis of hemophilia A
  • Patients starting efanesoctocog alfa treatment as per standard of care no more than one month prior to the enrollment date, for either on demand or prophylaxis. Patients starting efanesoctocog alfa treatment for a surgery event may also be enrolled only if the treatment is prescribed at enrollment.
  • Physician's decision to treat the patient with efanesoctocog alfa is made prior to and independently of participation in the study.
  • Signed and dated informed consent provided by the patient, or by the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric patients according to local regulations.

Exclusion criteria

Diagnosed with other known bleeding disorder

  • Participation in an investigational medicinal product trial at enrollment visit, or intake of an Investigational Medicinal Product within 3 months prior to inclusion in this study
  • Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥0.60 BU/mL

"The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial."

Treatment and study plan

Efanesoctocog Alfa BIVV001

Drug

Given per investigator's discretion

Other names: ALTUVIIIO

Primary outcomes

  1. Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds

    Time frame: Up to 5 years

    Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort

  2. Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds

    Time frame: Up to 5 years

    Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort

  3. Number of Target joint development, resolution and/or recurrence

    Time frame: Up to 5 years

    Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic coho

Secondary outcomes

  1. Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores

    Time frame: At 1, 2, 3, 4, 5 years

    HJHS total/domain scores will be reported for prophylactic cohorts (A1 ad A2)

  2. Annual Bleeding Rate (ABR) by type for treated bleeds

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (A1 and A2)

  3. ABR by type all (treated and untreated) bleeds

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (A1 and A2)

  4. ABR by location for treated bleeds

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (A1 and A2)

  5. ABR by location for all (treated and untreated) bleeds

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (A1 and A2)

  6. ABR for all bleeding episodes (including untreated bleeding episodes)

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (A1 and A2)

  7. Percentage of patients with zero joint bleeds

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (A1 and A2)

  8. Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (A1 and A2)

  9. Annualized injection frequency per participant (assessed by prescription) during the follow- up period

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (A1 and A2)

  10. Treatment adherence (%) as judged by the physician during the follow-up period

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (A1 and A2)

  11. Number of injections of efanesoctocog alfa to treat a bleeding episode.

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)

  12. Total dose of efanesoctocog alfa to treat a bleeding episode.

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)

  13. Number of injections of efanesoctocog alfa to treat a bleeding episode

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for On-demand cohort

  14. Total dose of efanesoctocog alfa to treat a bleeding episode

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for On-demand cohort

  15. Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for On-demand cohort

  16. Occurrence of a change in treatment regimen (on-demand to prophylactic or prophylactic to on-demand) at baseline and follow-up.

    Time frame: At 1, 2, 3, 4, 5 years

    Data will be reported for On-demand cohort

  17. Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores

    Time frame: At 1, 2, 3, 4 and 5 years

    HEAD-US total/domain scores will be reported for joint imaging sub cohort

  18. Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS)

    Time frame: At 1, 2, 3, 4 and 5 years

    Data will be reported for consenting participants from the joint imaging sub-cohort

  19. Change from baseline in synovial hypertrophy by change in mm thickness AND/OR HEAD-US synovitis domain, AND/OR by JADE MSKUS synovial hypertrophy +/- power doppler signal

    Time frame: At 6 months, 1, 2, 3, 4 and 5 years

    Data will be reported from the consenting participants from the joint imaging sub-cohort

  20. Occurrence of adverse events (AEs) and serious adverse events (SAEs)

    Time frame: Over 5 year period

    The safety and tolerability of efanesoctocog alfa over 5 years evaluated in all participants

  21. Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay.

    Time frame: Over 5 year period

    The safety and tolerability of efanesoctocog alfa over 5 years assessed in all participants

  22. Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) pain intensity 3a questionnaire (adults)

    Time frame: At 3 months, 6 months, 1, 2, 3, 4 and 5 years

    Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants

  23. Change from baseline in Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) 3.0 (boys ≤18 years old) questionnaire (including Parent Proxy)

    Time frame: At 1, 2, 3, 4 and 5 years

    Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants

  24. Change from baseline in the occurrence of inpatient and outpatient visits, and length of hospital stay related to hemophilia A

    Time frame: At 1, 2, 3, 4 and 5 years

    Healthcare resource use (HCRU) in efanesoctocog alfa related to hemophilia A over a 5-year period

  25. Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa.

    Time frame: Over a 5 year period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period

  26. Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor)

    Time frame: Over 5 year period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period

  27. Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)

    Time frame: Over 5 year period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period

  28. Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)

    Time frame: Over 5 year period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period

  29. Number of blood component transfusions used during perioperative period for surgery

    Time frame: Over 5 year Period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period

  30. Type of blood component transfusions used during perioperative period for surgery

    Time frame: Over 5 year period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period

  31. Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery

    Time frame: Over 5 year period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period

  32. Number of transfusions required for surgery (intraoperative and post-operative period)

    Time frame: Over 5 year period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period

  33. Duration of hospitalization (major, minor)

    Time frame: Over 5 years period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period.

  34. Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)

    Time frame: Over a 5 year period

    Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period for On-demand Cohort

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study title

Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A

Important dates

Study start
2023
Primary completion
2028
Study completion
2030
First posted
Jun 22, 2023
Registry last updated
Oct 2, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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