Clinical trial NCT06684314: A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
Official title: PROphylactic Efanesoctocog Alfa Therapy Evaluated for Critical Joint Health in Hemophilia A Treatment: The PROTECT-ALT Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hemophilia A
Keywords: Classic Hemophilia
Factor VIII Deficiency
Haemophilia
Hemophilia
Hemarthrosis
Blood Coagulation Disorders, Inherited
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
First posted: 2024-11-12
Enrollment: 100
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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.

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Change from baseline in annualized joint bleeding rate (AjBR) for treated and untreated bleeds
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Number of target joint development, resolution and/or recurrence
timeFrame: At 1, 2 ,3, 4 and 5 years
Secondary outcomes:
measure: Annualized bleeding rate (ABR) by type and location for treated and all (treated and untreated) bleeds
timeFrame: At 1, 2, 3, 4 and 5 years
measure: ABR for all bleeding episodes, including untreated bleeding episodes
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Percentage of participants with zero joint bleeds
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Number of injections and doses of efanesoctocog alfa to treat a bleeding episode
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Annualized injection frequency per participant (assessed by prescription) during the followup period
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Treatment adherence (%) as judged by the physician during the follow-up period
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Occurrence of a change in treatment regimen at baseline and follow-up
timeFrame: 5 years
measure: Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores in all joints
timeFrame: Every 6 months for 5 years
measure: Change from baseline in joint status on functional HJHS
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Number and percentage of joints with no prior joint damage at baseline, without joint damage on joint ultrasound
timeFrame: At 5 years
measure: Changes from baseline in HEAD-US synovitis domain
timeFrame: Every 6 months for 5 years
measure: Change from baseline in MRI score
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Number and percentage of participants without joint damage on MRI
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Number and percentage of joints with no prior joint damage at baseline, without joint damage on MRI
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa
timeFrame: 5 years
measure: Number of injections and dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
timeFrame: 5 years
measure: Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
timeFrame: 5 years
measure: Number and type of blood component transfusions used during perioperative period for surgery
timeFrame: 5 years
measure: Estimated blood loss (mL) during perioperative period for major surgery
timeFrame: 5 years
measure: Number of transfusions required for surgery (intraoperative and post-operative period)
timeFrame: 5 years
measure: Duration of hospitalization (major, minor)
timeFrame: 5 years
measure: Occurrence of adverse events (AEs), serious adverse events (SAEs) and adverse events of special interest (AESI)
timeFrame: 5 years
measure: Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay)
timeFrame: 5 years
measure: Change from baseline in HAL (haemophilia activities list) for adult participants only
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Change from baseline in PROMIS pain intensity 3a questionnaire for adult participants only
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Change from baseline in PROBE full questionnaire for adult participants only
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Change from baseline in Ped-HAL (haemophilia activities list) for adolescent participants only (≥ 12 to <18 years)
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Change from baseline in PROMIS pain intensity 1a questionnaire for adolescent participants only (≥ 12 to <18 years)
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Change from baseline in PROBE full questionnaire for adolescent participants only (≥ 12 to <18 years)
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Change from baseline in Ped-HAL (pediatric haemophilia activities list) for pediatric participants only (12 years and younger)
timeFrame: At 1, 2, 3, 4 and 5 years
measure: Number of hospitalizations excluding surgery and admission/discharge diagnoses
timeFrame: Change from baseline at 1, 2, 3, 4 and 5 years
measure: Number of hospitalization days
timeFrame: Change from baseline at 1, 2, 3, 4 and 5 years
measure: Number of intensive care unit (ICU) stays
timeFrame: Change from baseline at 1, 2, 3, 4 and 5 years
measure: Number of emergency room visits
timeFrame: Change from baseline at 1, 2, 3, 4 and 5 years
measure: Number of outpatient visits
timeFrame: Change from baseline at 1, 2, 3, 4 and 5 years
Other outcomes:
Locations:
city: Changhua
country: Taiwan
facility: Investigational Site Number : 1580008
city: Kaohsiung City
country: Taiwan
facility: Investigational Site Number : 1580009
city: Kaohsiung City
country: Taiwan
facility: Investigational Site Number : 1580010
city: Taichung
country: Taiwan
facility: Investigational Site Number : 1580005
city: Taichung
country: Taiwan
facility: Investigational Site Number : 1580006
city: Taichung
country: Taiwan
facility: Investigational Site Number : 1580007
city: Taipei City
country: Taiwan
facility: Investigational Site Number : 1580001
city: Taipei City
country: Taiwan
facility: Investigational Site Number : 1580003
city: Taipei City
country: Taiwan
facility: Investigational Site Number : 1580002
city: Taoyuan City
country: Taiwan
facility: Investigational Site Number : 1580004
Study officials:
References: