2022-502921-16-00已完成4 期
An 18-month low-interventional prospective, multicentre study to assess joint outcomes in patients with haemophilia A or B on prophylaxis with efmoroctocog alfa or eftrenonacog alfa
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 入组人数
- 247
- 试验地点
- 50
- 主要终点
- Change from baseline in total HEAD-US score up to month 18 (end of study - EOS)
研究概览
简要总结
To evaluate the overall joint status as detected by ultrasound (US) in haemophilia A and B patients treated with efmoroctocog alfa (rFVIIIFc) or eftrenonacog alfa (rFIXFc) prophylaxis over the 18-month study period.
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
- 是
入选标准
- •Age ≥ 6 years
- •Diagnosis of haemophilia A or B
- •Having at least 6 months documented pre-study treatment data regarding treatment prescriptions and bleeding episodes prior to the baseline visit
- •Previous treatment for haemophilia A or B with any marketed recombinant and/or plasma-derived FVIII or FIX concentrate for at least 6 months
- •Start of prophylactic treatment with rFVIIIFc or rFIXFc prior to study enrolment or latest at the baseline visit, in accordance with local regulations
- •Signed and dated informed consent provided by the patient, or the patient’s legally authorized representative for patients under the legal age. Assent should be obtained from paediatric patients in accordance with local regulations
- •To be eligible for the florio HAEMO sub-study a patient should have used florio HAEMO (a CE marked medical device used in routine clinical practice) for at least 3 months and must agree to have data collected from the florio HAEMO app by providing a separate informed consent or assent
排除标准
- •Any medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion
- •Prophylactic treatment with non-factor therapy during the 6 months prior to enrolment
- •Presence of factor VIII or FIX inhibitory antibodies (inhibitors) (≥0.60 Bethesda Units [BU]/mL) at the latest available inhibitor test
- •Enrolment in a concurrent clinical interventional study, or intake of an Investigational medicinal product, within 3 months prior to inclusion in this study
- •Foreseeable inability to cooperate with given instructions or study procedures
结局指标
主要结局
Change from baseline in total HEAD-US score up to month 18 (end of study - EOS)
Change from baseline in total HEAD-US score up to month 18 (end of study - EOS)
次要结局
- IPAQ-SF scores at baseline and 18 months
- Change from baseline in HEAD-US score for hypertrophic synovium at 6, 12 and 18 months
- Change from baseline in HEAD-US score for cartilage at 6, 12 and 18 months
- Change from baseline in HEAD-US score for bone at 6, 12 and 18 months
- Change from baseline in total HJHS at month 18 (EOS)
- Number and location of target joints at baseline, 6, 12 and 18 months
- Total Annualized bleeding rate (ABR), joint ABR, target joint ABR, traumatic/spontaneous ABR
- Patient-Reported Outcomes Measurement Information System (PROMIS) physical function/activity scores at baseline and 18 months
- PROMIS pain intensity and interference scores at baseline and 18 months
研究者
Ekaterina GRESKO
Scientific
Swedish Orphan Biovitrum AB (publ)
研究点 (50)
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