A Low-interventional Study to Evaluate Long-term Effectiveness of Real-world Prophylactic Treatment With Efanesoctocog Alfa on Joint Health in People With Haemophilia A (ALTITUDE)
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 发起方
- 入组人数
- 250
- 试验地点
- 29
- 主要终点
- Annualized joint bleeding rate (AjBR) over the prospective observation period.
研究概览
简要总结
The rationale for this study is to further understand and describe the long-term prophylactic effectiveness of efanesoctocog alfa in preventing joint bleeds in a real-life setting.
详细描述
This is a retrospective-prospective, low-interventional study to evaluate the long-term effectiveness of real-world prophylaxis with efanesoctocog alfa on joint health in people with haemophilia A. Participants in the study must be prescribed efanesoctocog alfa at the discretion of their physicians within 6 months prior to, or at the time of, enrolment. The decision to initiate treatment with efanesoctocog alfa shall be made independently from the decision to include the patients in the study. A broad haemophilia A population (e.g. all severities [mild to severe], all ages, patients with comorbidities and/or concomitant use of anticoagulant/antiplatelet drugs, patients with previous inhibitors to Factor FVIII [FVIII]) will be eligible to enrol. The study will require mandatory on-site visits with joint assessments. A retrospective data collection on patients' haemophilia, medical, and surgical history will be performed, including previous haemophilia treatment and treated bleeding episodes from 12 months prior to initiating treatment with efanesoctocog alfa until enrolment.
The study will start with the enrolment visit during a patient's routine visit to the hospital, to check whether the patient fulfils all the inclusion criteria and none of the exclusion criteria. Study participants will have yearly on-site visits for 24 months or 36 months, depending on the time of enrolment in relation to study closure. Patients will be followed in the study as long as they are treated with efanesoctocog alfa prophylaxis which would be at the physicians' discretion. Patients will also need to agree to document any bleeding episodes they may have during the study in a study-specific paper diary. Joint assessment, Haemophilia Joint Health Score (HJHS), and completion of Patient-reported outcomes (PROs) will be conducted during the on-site visits. For patients included in the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) group, ultrasound joint assessment will also be done at the yearly visits. The end of study (EoS) visit can be performed at the 24-month visit or the 36-month visit, depending on the time of enrolment.
The target is to enroll approximately 250 participants.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Male or female patient with a diagnosis of haemophilia A.
- •Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.
- •Prescribed with efanesoctocog alfa prophylactic treatment within 6 months prior to study enrolment or at the latest at the enrolment visit, in accordance with local regulations.
- •Must have received prophylactic treatment(s) with any haemophilia product(s) for at least 12 months prior to being prescribed with efanesoctocog alfa treatment.
- •Have documented pre-study treatment data on haemophilia prophylaxis prescriptions and on treated bleeding episodes from the 12-months prior to being prescribed efanesoctocog alfa treatment and until enrolment into this study.
- •Willing to adhere to the visit schedule and to undergo mandatory study assessment (HJHS, PROs).
- •Willingness and the ability of the patient or their legally designated representative to document all bleeding episodes, including any treatments for those episodes.
- •For HEAD-US subgroup: Accept to undergo annual ultrasound assessment on the index joints (ankles, elbows, knees).
排除标准
- •Acquired haemophilia A and other blood clotting disorders than hereditary haemophilia A.
- •Any positive FVIII inhibitor result (defined as inhibitor titre ≥0.6 Bethesda unit [BU]/mL) from the medical records in connection to the switch to efanesoctocog alfa until the enrolment visit.
- •Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), including for haemophilia prophylaxis, within 3 months prior to enrolment in this study.
- •Patient not suitable for participation, whatever the reason, as judged by the Investigator, e.g., patient is not able or willing to perform the study assessments.
研究组 & 干预措施
Study Treatment Group
干预措施: Efanesoctocog alfa (Drug)
结局指标
主要结局
Annualized joint bleeding rate (AjBR) over the prospective observation period.
时间窗: Enrolment to Month 12, 24 and 36
次要结局
- Patient Reported Outcome of Parent Proxy EuroQol 5-dimensions [EQ-5D-Y:1] score(Enrolment to Month 12, 24 and 36)
- Patient Reported Outcome of ACTIVLIM-Hemo.(Enrolment to Month 12, 24 and 36)
- Number of participants with occurrence of Serious Adverse Events (SAEs).(Enrolment to 36 months)
- Number of participants with occurrence of Adverse Events of Special Interest (AESIs).(Enrolment to 36 months)
- Annualized bleeding rate (ABR) over the prospective observation period.(Enrolment to Month 12, 24 and 36)
- Number of injections to control a bleeding event over the prospective observation period.(Enrolment to Month 12, 24 and 36)
- Total dose to control bleeding event over the prospective observation period.(Enrolment to Month 12, 24 and 36)
- Haemophilia Joint Health Score (HJHS) version 2.1(Enrolment to Month 12, 24 and 36)
- Target joint development on a patient and joint level over the prospective observation period.(Month 12, 24 and 36)
- Target joint resolution on a patient and joint level over the prospective observation period.(Month 12, 24 and 36)
- Target joint recurrency on a patient and joint level over the prospective observation period.(Month 12, 24 and 36)
- Annualised injection frequency over the prospective observation period.(Month 12, 24 and 36)
- Annualised efanesoctocog alfa consumption for prophylaxis and treatment of bleeds (excluding surgery) over the prospective observation period.(Month 12, 24 and 36)
- Reason(s) for any changes in prescribed frequency and/or dose of efanesoctocog alfa prophylaxis over the prospective observation period.(Enrolment to Month 12, 24 and 36)
- Change in ABR in the period of using efanesoctocog alfa vs prior haemophilia treatment.(Prospective period vs Retrospective period)
- Change in annualised injection frequency during the period on efanesoctocog alfa vs the period on prior haemophilia treatment.(Prospective period vs Retrospective period)
- Change in annualised consumption during the period on efanesoctocog alfa vs the period on prior haemophilia treatment.(Prospective period vs Retrospective period)
- Reason(s) for Switching to efanesoctocog alfa treatment.(Retrospective period (12 to 18 months prior to enrollment))
- Investigator or Surgeon assessment of patient's haemostatic response on the International Society on Thrombosis and Haemostasis (ISTH) 4-point response for surgical procedures scale (Intraoperative and postoperative).(Within 96 hours following surgery)
- Number of injections and dose administered to maintain haemostasis during perioperative period for minor and major surgery.(Enrolment to 36 Months)
- Total efanesoctocog alfa consumption during perioperative period for minor/major surgery.(Enrolment to 36 Months)
- Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)(Enrolment to Month 12, 24 and 36)
- Treatment preference survey(Month 12)
- Patient Reported Outcome (PRO) of Pain Intensity(Enrolment to Month 12, 24 and 36)
- Patient Reported Outcome (PRO) of Pain Interference(Enrolment to Month 12, 24 and 36)
- Patient Reported Outcome of 5-level EuroQol-5 dimensions [EQ-5D-5L] score(Enrolment to Month 12, 24 and 36)
- Patient Reported Outcome of youth EuroQol 5-dimensions [EQ-5D-Y] score(Enrolment to Month 12, 24 and 36)