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NCT Number: NCT06684314

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

This is a national, multicenter, retrospective/prospective, observational study in Taiwan designed to assess effectiveness, safety, and usage of efanesoctocog alfa prophylaxis treatment in hemophilia A participants. The data related to efanesoctocog alfa effectiveness, safety and usage will be recorded prospectively during routine visits for up to 5 years following enrollment initiation and the retrospective data will be collected at least 12 months and up to 24 months prior to efanesoctocog alfa initiation. Joint imaging data will be collected in centers performing Joint U/S and/or MRI (≥6 years old). At least 12 months of retrospective data will also be collected from medical records, as available. Prospectively collected data will be recorded at routine clinical visits during a five-year follow-up period. The end of study is defined as the last participant's last visit. No intervention will be administered, and no study related visits are required.

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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 Hemarthrosis Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Joint Diseases Musculoskeletal Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms

Sex eligibility

All sexes

Study type

Observational

Primary location

Investigational Site Number : 1580008, Changhua, Taiwan

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • Participants with all ages and diagnosis of moderate-severe hemophilia A without current and/or at least three years of un-detectable inhibitor (<0.6 BU)
  • Participants with moderate to severe hemophilia A as defined by FVIII level ≤ 5%
  • Participants starting efanesoctocog alfa prophylaxis treatment as per standard of care no more than three months prior to the enrollment date
  • Participants aged 6 years and older are able to undergo MRI examinations (sedation given, if necessary, and per investigator discretion)
  • Participants are able to undergo joint examinations
  • Physician's decision to treat the participant with efanesoctocog alfa is made prior to and independently of participation in the study
  • Signed and dated informed consent provided by the participant, or by the participant's legally acceptable representative for participants under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric participants according to local regulations

Exclusion criteria

  • Participants with coagulation disorders other than hemophilia A
  • Participants diagnosed with other known bleeding disorder
  • Participants currently receive factor therapy and have signs of decreased response to FVIII therapy
  • Participants with a baseline Radiological Pettersson score (PS) of greater than 6 for each individual ankle
  • Enrollment in another concurrent clinical interventional study, or intake of an Investigational Medicinal Product within 3 months prior to inclusion in this study
  • Pregnant female participants

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

Drug

This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practices.

Other names: ALTUVIIIO, BIVV001

Primary outcomes

  1. Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints

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

    The HJHS summarizes joint health by providing a clinical score in the domain of body structure and function (i.e., impairment), of the joints most commonly affected by bleeding in hemophilia: (left ankle, right ankle, left elbow, right elbow, left knee, right knee). The HJHS consists of assessments of swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain and strength for elbows, knees and ankles and a global gait score. A higher score indicates worse joint health.

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

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

    The AjBR is defined as the number of joint bleeding episodes occurring during the treatment period divided by the duration of the treatment period in days multiplied by 365.25. All types of joint bleeding episodes (spontaneous, traumatic, and type unknown) will be included in determining the annualized number.

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

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

Secondary outcomes

  1. Annualized bleeding rate (ABR) by type and location for treated and all (treated and untreated) bleeds

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

  2. ABR for all bleeding episodes, including untreated bleeding episodes

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

  3. Percentage of participants with zero joint bleeds

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

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

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

  5. Number of injections and doses of efanesoctocog alfa to treat a bleeding episode

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

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

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

  7. Annualized injection frequency per participant (assessed by prescription) during the followup period

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

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

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

  9. Occurrence of a change in treatment regimen at baseline and follow-up

    Time frame: 5 years

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

    Time frame: Every 6 months for 5 years

  11. Change from baseline in joint status on functional HJHS

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

  12. Number and percentage of joints with no prior joint damage at baseline, without joint damage on joint ultrasound

    Time frame: At 5 years

  13. Changes from baseline in HEAD-US synovitis domain

    Time frame: Every 6 months for 5 years

  14. Change from baseline in MRI score

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

  15. Number and percentage of participants without joint damage on MRI

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

  16. Number and percentage of joints with no prior joint damage at baseline, without joint damage on MRI

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

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

    Time frame: 5 years

  18. Number of injections and dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)

    Time frame: 5 years

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

    Time frame: 5 years

  20. Number and type of blood component transfusions used during perioperative period for surgery

    Time frame: 5 years

  21. Estimated blood loss (mL) during perioperative period for major surgery

    Time frame: 5 years

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

    Time frame: 5 years

  23. Duration of hospitalization (major, minor)

    Time frame: 5 years

  24. Occurrence of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESI)

    Time frame: 5 years

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

    Time frame: 5 years

  26. Change from baseline in HAL (haemophilia activities list) for adult participants only

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

    HAL contains 42 items across 7 domains: lying down/ sitting/ kneeling/ standing, functions of the legs, functions of the arms, use of transportation, self-care, household tasks and leisure activities and sports. Items are scored on a 6-point Likert scale ('impossible', 'always', 'usually', 'sometimes', 'almost never', 'never'), with a 'not applicable' option for some items. A summary score as well as component scores (upper extremity, basic lower extremity and complex lower extremity) can be calculated. The scores are converted to a normalized score from 0 to 100, where higher scores represent a better functional status.

  27. Change from baseline in PROMIS pain intensity 3a questionnaire for adult participants only

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

    The PROMIS Pain Intensity instrument assesses how much a person hurts. The Pain Intensity short forms are universal rather than disease specific. The 1a and the first two items within the PROMIS Scale- Pain Intensity 3a short form assesses pain intensity over the past seven days. The version 2.0 of Pain Intensity 3a instruments are available for adults (ages 18+), and the version v1.0 of Pediatric Pain Intensity 1a self-report (ages 8-17) will be used for this study.

  28. Change from baseline in PROBE full questionnaire for adult participants only

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

    The PROBE questionnaire assesses participant reported outcomes in people with hemophilia and people without a bleeding disorder. The PROBE questionnaire is comprised of four major sections, including demographic data, general health problems, hemophilia-related health problems and health-related quality of life. It combines generic and disease-specific outcomes in 29 questions.

  29. Change from baseline in Ped-HAL (haemophilia activities list) for adolescent participants only (≥ 12 to <18 years)

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

    PedHAL contains 53 items across 7 domains: sitting/ kneeling/ standing, functions of the legs, functions of the arms, use of transportation, self-care, household tasks and leisure activities and sports. It consists of a participant version (8-18 years) and parent version (4-17 years). Items are scored on a 6-point Likert scale ('impossible', 'always', 'usually', 'sometimes', 'almost never', 'never'), with a 'not applicable (N/A)' scoring option for all items. A summary score as well as domain scores can be calculated. The scores are converted to a normalized score from 0 to 100, where higher scores represent a better functional status.

  30. Change from baseline in PROMIS pain intensity 1a questionnaire for adolescent participants only (≥ 12 to <18 years)

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

    The PROMIS Pain Intensity instrument assesses how much a person hurts. The Pain Intensity short forms are universal rather than disease specific. The 1a and the first two items within the PROMIS Scale- Pain Intensity 3a short form assesses pain intensity over the past seven days. The version 2.0 of Pain Intensity 3a instruments are available for adults (ages 18+), and the version v1.0 of Pediatric Pain Intensity 1a self-report (ages 8-17) will be used for this study.

  31. Change from baseline in PROBE full questionnaire for adolescent participants only (≥ 12 to <18 years)

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

    The PROBE questionnaire assesses participant reported outcomes in people with hemophilia and people without a bleeding disorder. The PROBE questionnaire is comprised of four major sections, including demographic data, general health problems, hemophilia-related health problems and health-related quality of life. It combines generic and disease-specific outcomes in 29 questions.

  32. Change from baseline in Ped-HAL (pediatric haemophilia activities list) for pediatric participants only (12 years and younger)

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

    PedHAL contains 53 items across 7 domains: sitting/ kneeling/ standing, functions of the legs, functions of the arms, use of transportation, self-care, household tasks and leisure activities and sports. It consists of a participant version (8-18 years) and parent version (4-17 years). Items are scored on a 6-point Likert scale ('impossible', 'always', 'usually', 'sometimes', 'almost never', 'never'), with a 'not applicable (N/A)' scoring option for all items. A summary score as well as domain scores can be calculated. The scores are converted to a normalized score from 0 to 100, where higher scores represent a better functional status.

  33. Number of hospitalizations excluding surgery and admission/discharge diagnoses

    Time frame: Change from baseline at 1, 2, 3, 4 and 5 years

  34. Number of hospitalization days

    Time frame: Change from baseline at 1, 2, 3, 4 and 5 years

  35. Number of intensive care unit (ICU) stays

    Time frame: Change from baseline at 1, 2, 3, 4 and 5 years

  36. Number of emergency room visits

    Time frame: Change from baseline at 1, 2, 3, 4 and 5 years

  37. Number of outpatient visits

    Time frame: Change from baseline at 1, 2, 3, 4 and 5 years

Study contacts

Contact information is provided by the study sponsor or research team.

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

[email protected]

800-633-1610 ext. option 6

Sponsors and collaborators

Lead sponsor

Sanofi

Industry

Registry information

Official study title

PROphylactic Efanesoctocog Alfa Therapy Evaluated for Critical Joint Health in Hemophilia A Treatment: The PROTECT-ALT Study

Acronym: PROTECT-ALT

Important dates

Study start
2024
Primary completion
2031
Study completion
2031
First posted
Nov 12, 2024
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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