PROphylactic Efanesoctocog Alfa Therapy Evaluated for Critical Joint Health in Hemophilia A Treatment: The PROTECT-ALT Study
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- Sanofi
- 入组人数
- 100
- 试验地点
- 10
- 主要终点
- Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints
研究概览
简要总结
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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.
研究组 & 干预措施
Efanesoctocog alfa
Participants observed receiving treatment with efanesoctocog alfa for hemophilia A
干预措施: Efanesoctocog alfa (Drug)
结局指标
主要结局
Change from baseline in total scores of the Hemophilia Joint Health Score (HJHS) for all joints
时间窗: 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
时间窗: 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
时间窗: At 1, 2 ,3, 4 and 5 years
次要结局
- 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(At 1, 2, 3, 4 and 5 years)
- Change from baseline in PROMIS pain intensity 3a questionnaire for adult participants only(At 1, 2, 3, 4 and 5 years)
- Change from baseline in PROBE full questionnaire for adult participants only(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 outpatient visits(Change from baseline 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)(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)
- 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)
- Change from baseline in PROBE full questionnaire for adolescent participants only (≥ 12 to <18 years)(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)(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)