2024-512066-33-00招募中4 期
A 12-month, interventional, open-label, phase 4 study in Europe (SHINE) to investigate the course of synovial hypertrophy as detected by joint ultrasound and MRI in patients with haemophilia A on efanesoctocog alfa prophylaxis.
试验速览
- 阶段
- 4 期
- 状态
- 招募中
- 入组人数
- 35
- 试验地点
- 10
- 主要终点
- Joints with synovial hypertrophy at baseline (HEAD-US synovial hypertrophy domain score of 1 or 2) and at least 1 point decrease in HEAD-US synovial hypertrophy domain score at Month 12 (Yes/No)
研究概览
简要总结
To assess the improvement of existing synovial hypertrophy in joints of patients receiving efanesoctocog alfa prophylaxis
研究设计
- 分配方式
- Not Applicable
- 主要目的
- The End of Treatment (EoT)
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 65+ years（0-17 Years, 18-64 Years, 65+ Years）
- 接受健康志愿者
- 否
入选标准
- •Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and protocol. Parents’ or legally designated representatives’ consent is required for patients who are <18 years of age or unable to give consent, or as applicable per local laws. Patients who are <18 years of age should provide assent in addition to the parents’/legally designated representatives’ consent, if appropriate.
- •Male or female patients who are ≥12 years of age and diagnosed with moderate or severe haemophilia A (defined as ≤5% of normal FVIII clotting activity) at the time of signing the ICF.
- •A female patient is eligible to participate if she is not pregnant at enrolment and does not plan to become pregnant during the study. A woman of child-bearing potential (WOCBP) must have a negative highly sensitive serum pregnancy test at the Screening Visit.
- •Must have received prophylactic treatment per local label with any marketed FVIII product or emicizumab for ≥12 months prior to the Baseline Visit.
- •Have at least one eligible index joint (ankle, elbow, knee).
- •Have 12 months of documented pre-study treatment data on haemophilia prescriptions and on treated bleeding episodes prior to the Baseline Visit.
- •Willingness and the ability of the patient or their legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study.
排除标准
- •Blood clotting disorders other than haemophilia A
- •Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.
- •Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), within 3 months prior to inclusion in the study.
- •Already on efanesoctocog alfa treatment
- •Positive inhibitor result (assessed by local laboratory) from the Screening Visit, defined as ≥0.6 Bethesda units (BU)/mL.
- •History of inhibitors without successful immune tolerance induction (ITI) • Successful ITI is defined as: • Negative inhibitor titer (<0.6 BU/mL) • FVIII recovery > 66% of expected • FVIII half-life ≥ 6 hours
- •ITI performed within the last 2 years prior to the Baseline Visit.
- •Currently receiving treatment with any of the prohibited concomitant medications, as specified by the protocol.
- •Planned major orthopaedic procedure in any eligible index joint during the course of the study.
- •Patients are not eligible for participation in the study if they cannot undergo MRI assessments at the Baseline Visit.
- •Patients with known hypersensitivity to the active substance or to any of the excipients.
结局指标
主要结局
Joints with synovial hypertrophy at baseline (HEAD-US synovial hypertrophy domain score of 1 or 2) and at least 1 point decrease in HEAD-US synovial hypertrophy domain score at Month 12 (Yes/No)
Joints with synovial hypertrophy at baseline (HEAD-US synovial hypertrophy domain score of 1 or 2) and at least 1 point decrease in HEAD-US synovial hypertrophy domain score at Month 12 (Yes/No)
次要结局
- Joints with no synovial hypertrophy at baseline (HEAD-US synovial hypertrophy domain score 0) and at least 1 point increase in HEAD-US synovial hypertrophy domain score at Month 6 or Month 12 (Yes/No)
- Distribution of joint HEAD-US synovial hypertrophy domain scores 0/1/2 at baseline and at Months 6 and 12
- Change from baseline in total/domain scores of HEAD-US per patient and per joint at Months 6 and 12
- Change from baseline in total/domain scores of International Prophylaxis Study Group (IPSG) MRI per patient and per joint at Month 12
- Change from baseline in total/domain scores of HJHS per patient and per joint at Month 12
- Patients with improved, unchanged, or worsened total/domain HEAD-US/MRI/HJHS scores at Month 12 from baseline
- Changes in PROs from baseline to Month 6 and Month 12 (assessed by 5- level EuroQol-5 dimensions [EQ-5D-5L], Patient-Reported Outcomes Measurement Information System [PROMIS] pain intensity, PROMIS pain interference, and PROMIS physical function)
- Patient-reported treatment preference at Month 12 (assessed with a questionnaire and exit interview)
- Change in ABR and annualized joint bleeding rate (AjBR) (spontaneous, traumatic) based on treated bleeds from the retrospective data collection period to on-study period
- Patients with target joint resolution or development from baseline to Month 12
- The occurrence of treatment-emergent adverse events (TEAEs) leading to treatment discontinuation, serious TEAEs, and adverse events of special interest (AESIs)
研究者
Contact point
Scientific
Swedish Orphan Biovitrum AB (publ)
研究点 (10)
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