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

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

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.

Study Overview

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

• Study Type: Observational
• Enrollment (Estimated): 100

Contacts and Locations

• Study Contact: Name: Trial Transparency email recommended (Toll free for US & Canada); Phone Number: option 6 800-633-1610; Email: contact-us@sanofi.com

Study Locations

• Taiwan
  • Changhua, Taiwan, 500
    • Recruiting
    • Investigational Site Number : 1580008
  • Kaohsiung City, Taiwan, 807
    • Recruiting
    • Investigational Site Number : 1580009
  • Kaohsiung City, Taiwan, 83301
    • Recruiting
    • Investigational Site Number : 1580010
  • Taichung, Taiwan, 40447
    • Recruiting
    • Investigational Site Number : 1580006
  • Taichung, Taiwan, 40705
    • Recruiting
    • Investigational Site Number : 1580007
  • Taichung, Taiwan, 40201
    • Recruiting
    • Investigational Site Number : 1580005
  • Taipei, Taiwan, 100
    • Recruiting
    • Investigational Site Number : 1580001
  • Taipei, Taiwan, 110
    • Recruiting
    • Investigational Site Number : 1580003
  • Taipei, Taiwan, 114
    • Recruiting
    • Investigational Site Number : 1580002
  • Taoyuan, Taiwan, 33305
    • Recruiting
    • Investigational Site Number : 1580004

Participation Criteria

Eligibility Criteria

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

• Sampling Method: Non-Probability Sample

Study Population

Participants from medical centers in Taiwan will be enrolled in the study after the introduction of efanesoctocog alfa for the treatment of hemophilia A. Eligible participants will include all ages, sexes and moderate to severe hemophilia A severities under efanesoctocog alfa prophylaxis treatment and able to undergo joint and/or MRI examinations during routine clinical practice.

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Efanesoctocog alfa; Participants observed receiving treatment with efanesoctocog alfa for hemophilia A	Drug: Efanesoctocog alfa; This study will not administer any treatment, only observe the treatment as prescribed in real-world clinical practices.; Other Names: BIVV001; ALTUVIIIO

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints	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.	At 1, 2, 3, 4 and 5 years
Change from baseline in annualized joint bleeding rate (AjBR) for treated and untreated bleeds	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.	At 1, 2, 3, 4 and 5 years
Number of target joint development, resolution and/or recurrence		At 1, 2 ,3, 4 and 5 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized bleeding rate (ABR) by type and location for treated and all (treated and untreated) bleeds		At 1, 2, 3, 4 and 5 years
ABR for all bleeding episodes, including untreated bleeding episodes		At 1, 2, 3, 4 and 5 years
Percentage of participants with zero joint bleeds		At 1, 2, 3, 4 and 5 years
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa		At 1, 2, 3, 4 and 5 years
Number of injections and doses of efanesoctocog alfa to treat a bleeding episode		At 1, 2, 3, 4 and 5 years
Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period		At 1, 2, 3, 4 and 5 years
Annualized injection frequency per participant (assessed by prescription) during the followup period		At 1, 2, 3, 4 and 5 years
Treatment adherence (%) as judged by the physician during the follow-up period		At 1, 2, 3, 4 and 5 years
Occurrence of a change in treatment regimen at baseline and follow-up		5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores in all joints		Every 6 months for 5 years
Change from baseline in joint status on functional HJHS		At 1, 2, 3, 4 and 5 years
Number and percentage of joints with no prior joint damage at baseline, without joint damage on joint ultrasound		At 5 years
Changes from baseline in HEAD-US synovitis domain		Every 6 months for 5 years
Change from baseline in MRI score		At 1, 2, 3, 4 and 5 years
Number and percentage of participants without joint damage on MRI		At 1, 2, 3, 4 and 5 years
Number and percentage of joints with no prior joint damage at baseline, without joint damage on MRI		At 1, 2, 3, 4 and 5 years
Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa		5 years
Number of injections and dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)		5 years
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)		5 years
Number and type of blood component transfusions used during perioperative period for surgery		5 years
Estimated blood loss (mL) during perioperative period for major surgery		5 years
Number of transfusions required for surgery (intraoperative and post-operative period)		5 years
Duration of hospitalization (major, minor)		5 years
Occurrence of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESI)		5 years
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay)		5 years
Change from baseline in HAL (haemophilia activities list) for adult participants only	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.	At 1, 2, 3, 4 and 5 years
Change from baseline in PROMIS pain intensity 3a questionnaire for adult participants only	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.	At 1, 2, 3, 4 and 5 years
Change from baseline in PROBE full questionnaire for adult participants only	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.	At 1, 2, 3, 4 and 5 years
Change from baseline in Ped-HAL (haemophilia activities list) for adolescent participants only (≥ 12 to <18 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.	At 1, 2, 3, 4 and 5 years
Change from baseline in PROMIS pain intensity 1a questionnaire for adolescent participants only (≥ 12 to <18 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.	At 1, 2, 3, 4 and 5 years
Change from baseline in PROBE full questionnaire for adolescent participants only (≥ 12 to <18 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.	At 1, 2, 3, 4 and 5 years
Change from baseline in Ped-HAL (pediatric haemophilia activities list) for pediatric participants only (12 years and younger)	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.	At 1, 2, 3, 4 and 5 years
Number of hospitalizations excluding surgery and admission/discharge diagnoses		Change from baseline at 1, 2, 3, 4 and 5 years
Number of hospitalization days		Change from baseline at 1, 2, 3, 4 and 5 years
Number of intensive care unit (ICU) stays		Change from baseline at 1, 2, 3, 4 and 5 years
Number of emergency room visits		Change from baseline at 1, 2, 3, 4 and 5 years
Number of outpatient visits		Change from baseline at 1, 2, 3, 4 and 5 years

Collaborators and Investigators

• Sponsor: Sanofi

Study record dates

Study Major Dates

• Study Start (Actual): November 11, 2024
• Primary Completion (Estimated): August 28, 2031
• Study Completion (Estimated): August 28, 2031

Study Registration Dates

• First Submitted: November 8, 2024
• First Submitted That Met QC Criteria: November 8, 2024
• First Posted (Actual): November 12, 2024

Study Record Updates

• Last Update Posted (Estimated): October 2, 2025
• Last Update Submitted That Met QC Criteria: October 1, 2025
• Last Verified: October 1, 2025

More Information

Terms related to this study

• Keywords: Hematologic Diseases; Genetic Diseases, Inborn; Haemophilia; Hemophilia; Hemorrhagic Disorders; Hemarthrosis; Factor VIII Deficiency; Coagulation Protein Disorders; Blood Coagulation Disorders, Inherited; Classic Hemophilia
• Additional Relevant MeSH Terms: Musculoskeletal Diseases; Pathologic Processes; Joint Diseases; Hemorrhage; Blood Coagulation Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Pathological Conditions, Signs and Symptoms; Hemic and Lymphatic Diseases; Genetic Diseases, Inborn; Hemophilia A; Hematologic Diseases; Hemorrhagic Disorders; Blood Coagulation Disorders, Inherited; Hemarthrosis; Coagulation Protein Disorders; BIVV001
• Other Study ID Numbers: OBS18067; U1111-1308-9835 (Registry Identifier: WHO ICTRP)

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 Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No