Health Related Quality of Life of Youth and Young Adults With Haemophilia A Treated With Efmoroctocog Alfa in Russia
试验速览
- 阶段
- 4 期
- 状态
- 已完成
- 发起方
- 入组人数
- 30
- 试验地点
- 1
- 主要终点
- Health-related quality of life
研究概览
简要总结
Research question:
Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis?
A 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations.
详细描述
Quality of life is among the key factors when medical decision about management of haemophilia is taken. There are no scientific data on the HRQoL in patients after switching from Standard Half Life (SHL) FVIII prophylaxis to efmoroctocog alfa (Extended Half Life, EHL) prophylaxis in Russia.
It will be a 12-month prospective open-label, single-arm multicentre study. Evaluation of parameters will be carried out on the backdrop of patient treatment in the settings of routine medical practice. No medical examinations/ procedures/ treatment(s) on the top of regular medical practice are planned, except fixed time of examinations. Providing the centers/ patients with the study drug (efmoroctocog alfa) may be considered as interventional component, whereas all administrations should be in accordance with Instruction on Medical Use approved by Russian Ministry of Health.
Research question:
Whether there are the changes in quality of life in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis?
No hypothesis is formulated for this exploratory study. The obtained data will be used to assess real world data on quality of life and clinical outcomes in patients with haemophilia A after switching from SHL FVIII prophylaxis to efmoroctocog alfa prophylaxis in routine medical practice in Russia
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Prevention
- 盲法
- None
入排标准
- 年龄范围
- 12 Years 至 25 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Signed consent form.
- •Adults, adolescents and children (12-25 years old) with haemophilia A with ABR ≥ 2 on at least one-year SHL FVIII prophylaxis.
排除标准
- •Patients who have had hypersensitivity reactions to efmoroctocog alfa or other constituents of the product.
- •History of Factor VIII inhibitors.
- •Patients who have other haemostatic disorders.
- •Patients participating in interventional studies.
研究组 & 干预措施
Patients receiving Efmoroctocog alfa
For long term prophylaxis, the recommended starting dose is 50 IU of factor VIII per kg body weight at intervals of 3 to 5 days. The dose may be adjusted based on patient response in the range of 25 to 65 IU/kg. In some cases, especially in younger patients, shorter dosage intervals or higher doses may be necessary.
干预措施: Efmoroctocog Alfa Injection [Eloctate] (Drug)
结局指标
主要结局
Health-related quality of life
时间窗: Month 12
Haemophilia Adult Quality of Life Questionnaire (Haem-A-QoL) - for patients of 18-25 y.o.; total score, domain scores. or Haemophilia-specific Quality of Life assessment for children and adolescents Short Form (Haemo-QoL SF) - for patients 12-17 y.o.; total score, domain scores.
次要结局
- Spontaneous bleedings - localisation(Month 1 - Month 3 - Month 6 - Month 12)
- Administration of efmorococtog alfa for correction of bleedings for Surgery/ Invasive Procedures(Month 1 - Month 3 - Month 6 - Month 12)
- Hospitalisations(Month 1 - Month 3 - Month 6 - Month 12)
- Factor VIII Activity(Day 0 - Month 1 - Month 3 - Month 6 - Month 12)
- Previous treatment/ prophylaxis of haemophilia(Day 0 - Month 1 - Month 3 - Month 6 - Month 12)
- Spontaneous bleedings(Month 1 - Month 3 - Month 6 - Month 12)
- Post-traumatic bleedings - localisation(Month 1 - Month 3 - Month 6 - Month 12)
- Surgery/ Invasive Procedures: major(Month 1 - Month 3 - Month 6 - Month 12)
- Hemophilia Joint Health Score(Day 0 - Month 6 - Month 12)
- Target Joints(Day 0 - Month 6 - Month 12)
- Administration of efmorococtog alfa for correction of bleedings(Month 1 - Month 3 - Month 6 - Month 12)
- Hospitalisations: duration(Month 1 - Month 3 - Month 6 - Month 12)
- Administration of efmoroctocog alfa(Day 0 - Month 1 - Month 3 - Month 6 - Month 12)
- Post-traumatic bleedings(Month 1 - Month 3 - Month 6 - Month 12)
- Surgery/ Invasive Procedures: minor(Month 1 - Month 3 - Month 6 - Month 12)
- Ultrasound Scoring(Day 0 - Month 6 - Month 12)
- Factor VIII Inhibitor Activity(Day 0 - Month 1 - Month 3 - Month 6 - Month 12)
- Administration of efmoroctocog alfa: factor consumption(Day 0 - Month 1 - Month 3 - Month 6 - Month 12)