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

October 1, 2025 updated by: Sanofi

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

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
100

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.
  • 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

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
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.
At 1, 2, 3, 4 and 5 years
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.
At 1, 2, 3, 4 and 5 years
Number of target joint development, resolution and/or recurrence
Time Frame: At 1, 2 ,3, 4 and 5 years
At 1, 2 ,3, 4 and 5 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
At 1, 2, 3, 4 and 5 years
ABR for all bleeding episodes, including untreated bleeding episodes
Time Frame: At 1, 2, 3, 4 and 5 years
At 1, 2, 3, 4 and 5 years
Percentage of participants with zero joint bleeds
Time Frame: At 1, 2, 3, 4 and 5 years
At 1, 2, 3, 4 and 5 years
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa
Time Frame: At 1, 2, 3, 4 and 5 years
At 1, 2, 3, 4 and 5 years
Number of injections and doses of efanesoctocog alfa to treat a bleeding episode
Time Frame: At 1, 2, 3, 4 and 5 years
At 1, 2, 3, 4 and 5 years
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
At 1, 2, 3, 4 and 5 years
Annualized injection frequency per participant (assessed by prescription) during the followup period
Time Frame: At 1, 2, 3, 4 and 5 years
At 1, 2, 3, 4 and 5 years
Treatment adherence (%) as judged by the physician during the follow-up period
Time Frame: At 1, 2, 3, 4 and 5 years
At 1, 2, 3, 4 and 5 years
Occurrence of a change in treatment regimen at baseline and follow-up
Time Frame: 5 years
5 years
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
Every 6 months for 5 years
Change from baseline in joint status on functional HJHS
Time Frame: At 1, 2, 3, 4 and 5 years
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
Time Frame: At 5 years
At 5 years
Changes from baseline in HEAD-US synovitis domain
Time Frame: Every 6 months for 5 years
Every 6 months for 5 years
Change from baseline in MRI score
Time Frame: At 1, 2, 3, 4 and 5 years
At 1, 2, 3, 4 and 5 years
Number and percentage of participants without joint damage on MRI
Time Frame: At 1, 2, 3, 4 and 5 years
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
Time Frame: At 1, 2, 3, 4 and 5 years
At 1, 2, 3, 4 and 5 years
Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa
Time Frame: 5 years
5 years
Number of injections and dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Time Frame: 5 years
5 years
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
Time Frame: 5 years
5 years
Number and type of blood component transfusions used during perioperative period for surgery
Time Frame: 5 years
5 years
Estimated blood loss (mL) during perioperative period for major surgery
Time Frame: 5 years
5 years
Number of transfusions required for surgery (intraoperative and post-operative period)
Time Frame: 5 years
5 years
Duration of hospitalization (major, minor)
Time Frame: 5 years
5 years
Occurrence of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESI)
Time Frame: 5 years
5 years
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay)
Time Frame: 5 years
5 years
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.
At 1, 2, 3, 4 and 5 years
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.
At 1, 2, 3, 4 and 5 years
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.
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)
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.
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)
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.
At 1, 2, 3, 4 and 5 years
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.
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)
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.
At 1, 2, 3, 4 and 5 years
Number of hospitalizations excluding surgery and admission/discharge diagnoses
Time Frame: Change from baseline at 1, 2, 3, 4 and 5 years
Change from baseline at 1, 2, 3, 4 and 5 years
Number of hospitalization days
Time Frame: Change from baseline at 1, 2, 3, 4 and 5 years
Change from baseline at 1, 2, 3, 4 and 5 years
Number of intensive care unit (ICU) stays
Time Frame: Change from baseline at 1, 2, 3, 4 and 5 years
Change from baseline at 1, 2, 3, 4 and 5 years
Number of emergency room visits
Time Frame: Change from baseline at 1, 2, 3, 4 and 5 years
Change from baseline at 1, 2, 3, 4 and 5 years
Number of outpatient visits
Time Frame: Change from baseline at 1, 2, 3, 4 and 5 years
Change from baseline at 1, 2, 3, 4 and 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Sanofi

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
November 11, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
August 28, 2031

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
August 28, 2031

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
November 8, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 8, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 12, 2024

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 2, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 1, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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