NCT05817812已完成3 期
A Phase 3b Open-label Study Evaluating Physical Activity and Joint Health in Previously Treated Patients ≥12 Years With Severe Haemophilia A Treated With Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (Efanesoctocog Alfa) for 24 Months
Swedish Orphan Biovitrum30 个研究点 分布在 14 个国家目标入组 93 人开始时间: 2023年7月31日最近更新:
适应症
干预措施
相关药物
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- 入组人数
- 93
- 试验地点
- 30
- 主要终点
- Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring
研究概览
简要总结
FREEDOM is a multicentre, open-label, single-arm, phase 3b study in Europe that aims to enrol approximately 90 previously treated severe haemophilia A patients aged ≥12 years, currently on prophylaxis. After a run-in period of 30-45 days, patients will receive efanesoctocog alfa prophylaxis, 50 IU/kg once-weekly for 24 months (additional preventive dose not permitted). An activity tracker and an electronic patient diary will be used to collect data on physical activity, bleeds, factor dosing, pain, and injuries from screening throughout the study.
The primary objective is to describe changes in physical activities over 24 months on efanesoctocog alfa prophylaxis, with a primary endpoint of change from baseline in International Physical Activity Questionnaire (IPAQ) at month 24.
Secondary objectives include relationship between physical activity and other variables (bleeds, joint status, pain, injuries, and quality of life); changes in joint status as assessed by HEAD-US, HJHS and MRI; occurrence of bleeds, injuries, pain. Safety and tolerability of efanesoctocog alfa will also be evaluated.
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
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Previous treatment for haemophilia A with any marketed recombinant and/or plasma-derived FVIII for at least 150 EDs.
- •Having received prophylactic treatment with any marketed recombinant and/or plasma FVIII or emicizumab per local label for at least 12 months preceding enrolment.
- •Having 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and 6 months data on bleeding episodes prior to baseline visit.
- •Willingness and ability to complete training in the use of the study ePD and to use the ePD in their own smartphone throughout the study.
- •Willingness and ability to use the activity tracker provided by the sponsor to measure physical activity and heart rate.
- •Be able and willing to administer efanesoctocog alfa intravenously at home.
排除标准
- •Serious musculoskeletal and/or neurological impairment limiting the mobility and the physical ability to a degree that makes the patient unsuitable for the study as judged by the investigator.
- •Other known coagulation disorder(s) in addition to haemophilia A.
- •History and/or current positive inhibitor test defined as ≥0.6 BU/mL.
- •Treatment with NSAIDs above the maximum dose specified in the prescribing information within 2 weeks prior to screening.
- •Systematic treatment within 12 weeks prior to screening with chemotherapy and/or other immunosuppressive drugs.
- •Treatment with an investigational product within 30 days or 5.5 half-lives prior to screening, whichever is longer.
- •Major surgery within 12 weeks prior to screening or planned major orthopaedic surgery to occur during the study.
- •At baseline visit, patients who have not been compliant in using the activity tracker.
研究组 & 干预措施
Efanesoctocog alfa
Experimental
All patients will be treated intravenously once weekly with 50 IU/kg efanesoctocog alfa
干预措施: Efanesoctocog alfa (Drug)
结局指标
主要结局
Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring
时间窗: Baseline and month 24
The IPAQ is a validated, 7-day recall questionnaire measuring current levels of physical activity. Scoring of the IPAQ results in a continuous variable in the form of total MET-minutes per week. Higher scores means higher physical activity level.
次要结局
- Mean value of frequency of workouts(Per 6-month period)
- Mean value of duration of workouts(Per 6-month period)
- Change in patient reported intensity of workouts(Run-in month, month 12 and 24)
- Change in mean daily minutes in physical activity(Run-in month, month 12 and 24)
- Change in frequency of workouts(Run-in month, month 12 and 24)
- Occurrence of pain in relation to workouts(Baseline to month 24)
- Change in type of workouts(Run-in month, month 12 and 24)
- Change in duration of workouts(Run-in month, month 12 and 24)
- Change in tracker recorded intensity of workouts(Run-in month, month 12 and 24)
- Mean value of patient and tracker reported type of workouts(Per 6-month period)
- Mean daily number of steps(Per 6-month period)
- Occurrence of bleeds in relation to workouts(Baseline to month 24)
- Target joint recurrence (≥3 spontaneous bleeds in a single joint within any consecutive 6-month period after target joint resolution)(Month 6, 12, 18, 24)
- Number of injections and dose to treat a bleeding episode(Baseline to month 24)
- Number of non-study medical care encounters(From baseline to month 24)
- International Physical Activity Questionnaire (IPAQ) score(Baseline, month 6, 12, 18 and 24)
- Mean daily minutes of physical activity(Per 6-month period)
- Mean value of patient reported intensity of workouts(Per 6-month period)
- Mean value of tracker recorded intensity of workouts(Per 6-month period)
- Occurrence of bleeding episodes impacting daily activity(Baseline to month 24)
- Target joint resolution ( ≤2 bleeds into the joint within a consecutive 12-month period the joint is no longer considered a target joint)(Month 6, 12, 18, 24)
- Change from baseline in International Physical Activity Questionnaire (IPAQ) scoring(Baseline and month 12)
- Change in mean daily number of steps(Run-in month, month 12 and 24)
- Change from baseline in Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)(Baseline, month 12 and 24)
- Annualized bleeding rate (ABR)(Baseline to month 24)
- Pain reported via electronic patient diary(Baseline to month 24)
- The occurrence of adverse events and serious adverse events(From screening to month 24)
- The occurrence of thrombotic and embolic events(From screening to month 24)
- Achieving WHO-recommended levels of MVPA(Run-in month, month 12 and 24)
- Occurrence of injuries in relation to workouts(Baseline to month 24)
- Change from baseline in haemophilic arthropathy assessed by the International Prophylaxis Study Group (IPSG) Magnetic Resonance Imaging (MRI) scale(Baseline, month 24)
- Pain interference reported in the Patient-Reported Outcomes Measurement Information System (PROMIS) scale - Pain interference 6a(Baseline, month 6, 12, 18, 24)
- Assessment of treatment preference(24 months)
- Development of inhibitors (neutralizing antibodies directed against FVIII)(Baseline, month 6, 12, 18, 24)
- Change from baseline in Haemophilia Joint Health Score (HJHS) score(Baseline, month 6, 12, 18, 24)
- Target joint development (three or more spontaneous bleeds into a single joint within a consecutive 6-month period)(Month 6, 12, 18, 24)
- Number of bleeding episodes(Per 6-month period and cumulative)
- Total annualized efanesoctocog alfa consumption(Baseline to month 24)
- Pain intensity reported in the Patient-Reported Outcomes Measurement Information System (PROMIS) Scale - Pain Intensity 3a(Baseline, month 6, 12, 18, 24)
- Quality of life assessed by EuroQoL 5-dimension 5-level (EQ-5D-5L)(Baseline, month 6, 12, 18, 24)
- The occurrence of clinically significant changes(Baseline, month 6, 12, 18, 24)
- Duration of non-study medical care encounters(From baseline to month 24)
研究者
研究点 (30)
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