A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A in Taiwan
- Registration Number
- NCT06684314
- Lead Sponsor
- Sanofi
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 120
- 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
- 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 PS score of greater than 6 in both ankles for each joint
- 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 & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Efanesoctocog alfa Efanesoctocog alfa Participants observed receiving treatment with efanesoctocog alfa for hemophilia A
- Primary Outcome Measures
Name Time Method Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints 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.
Change from baseline in annualized joint bleeding rate (AjBR) for treated and untreated bleeds 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.
Number of target joint development, resolution and/or recurrence At 1, 2 ,3, 4 and 5 years
- Secondary Outcome Measures
Name Time Method Change from baseline in HAL (haemophilia activities list) for adult participants only 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.
Change from baseline in PROBE full questionnaire for adult participants only 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.
Number and percentage of participants without joint damage on MRI 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 Annualized bleeding rate (ABR) by type and location for treated and all (treated and untreated) bleeds 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 Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa 5 years Change from baseline in PROMIS pain intensity 3a questionnaire for adult participants only 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.
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 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 Haem-A-QoL questionnaire for adult participants only At 1, 2, 3, 4 and 5 years The HAEM-A-QoL questionnaire, which is designed for adults and participants ≥ 17 years of age, is composed of 10 dimensions, which are "physical health", "feelings", "view", "sport and leisure time", "work and school", "dealing", "treatment", "future", "family planning", "relationship", with a total of 46 items.
Change from baseline in Haemo-QoL (kid) questionnaire for pediatric participants only (12 years and younger) At 1, 2, 3, 4 and 5 years The Haemo-QoL questionnaires are designed to assess the quality of life of children with hemophilia, focusing on children with hemophilia aged 4-16 years. The pediatric Haemo-QoL is designed to give different versions of the questionnaire depending on the age of the participant \[4-7 years (Children I), 8-12 years (Children II) and 13-16 years (Children III)\], and the questionnaire is composed of 8-12 dimensions, which are "physical health", "feelings", "attitudes", "family", "friends", "perceived support", "other persons", "sports and school", "dealing with the disease", "treatment", "future", "relationships".
Change from baseline in Haem-A-QoL or Haemo-QoL questionnaire based on age for adolescent participants only (≥ 12 to <18 years) At 1, 2, 3, 4 and 5 years The HAEM-A-QoL questionnaire, which is designed for adults and participants ≥ 17 years of age, is composed of 10 dimensions, which are "physical health", "feelings", "view", "sport and leisure time", "work and school", "dealing", "treatment", "future", "family planning", "relationship", with a total of 46 items.
The Haemo-QoL questionnaires are designed to assess the quality of life of children with hemophilia, focusing on children with hemophilia aged 4-16 years. The pediatric Haemo-QoL is designed to give different versions of the questionnaire depending on the age of the participant \[4-7 years (Children I), 8-12 years (Children II) and 13-16 years (Children III)\], and the questionnaire is composed of 8-12 dimensions, which are "physical health", "feelings", "attitudes", "family", "friends", "perceived support", "other persons", "sports and school", "dealing with the disease", "treatment", "future", "relationships".Change from baseline in Ped-HAL (haemophilia activities list) for adolescent participants only (≥ 12 to <18 years) 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.
Change from baseline in PROMIS pain intensity 1a questionnaire for adolescent participants only (≥ 12 to <18 years) 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.
Change from baseline in PROBE full questionnaire for adolescent participants only (≥ 12 to <18 years) 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.
Change from baseline in Ped-HAL (pediatric haemophilia activities list) for pediatric participants only (12 years and younger) 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.
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
Trial Locations
- Locations (8)
Investigational Site Number : 1580001
🇨🇳Taipei City, Taiwan
Investigational Site Number : 1580002
🇨🇳Taipei City, Taiwan
Investigational Site Number : 1580004
🇨🇳Taoyuan City, Taiwan
Investigational Site Number : 1580008
🇨🇳Changhua, Taiwan
Investigational Site Number : 1580005
🇨🇳Taichung, Taiwan
Investigational Site Number : 1580003
🇨🇳Taipei City, Taiwan
Investigational Site Number : 1580009
🇨🇳Kaohsiung City, Taiwan
Investigational Site Number : 1580006
🇨🇳Taichung, Taiwan