Health Related Quality of Life of Youth and Young Adults With Haemophilia A
- Conditions
- Hemophilia A
- Interventions
- Drug: Efmoroctocog Alfa Injection [Eloctate]
- Registration Number
- NCT04728217
- Lead Sponsor
- The League of Clinical Research, Russia
- Brief Summary
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.
- Detailed Description
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
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 30
- 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.
- 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.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description Patients receiving Efmoroctocog alfa Efmoroctocog Alfa Injection [Eloctate] 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.
- Primary Outcome Measures
Name Time Method Health-related quality of life 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.
- Secondary Outcome Measures
Name Time Method Spontaneous bleedings - localisation Month 1 - Month 3 - Month 6 - Month 12 Localisation of spontaneous bleedings: joints, muscles, subcutaneous/ submucosal, abdominal/ gastrointestinal, retroperitoneal, genitourinary, CNS, pharyngeal, other.
Administration of efmorococtog alfa for correction of bleedings for Surgery/ Invasive Procedures Month 1 - Month 3 - Month 6 - Month 12 Dose of efmorococtog alfa: IU.
Hospitalisations Month 1 - Month 3 - Month 6 - Month 12 Number from previous visit.
Factor VIII Activity Day 0 - Month 1 - Month 3 - Month 6 - Month 12 Factor VIII Activity (before injection; 30 min after injection), clotting assays (one stage): IU/dL.
Previous treatment/ prophylaxis of haemophilia Day 0 - Month 1 - Month 3 - Month 6 - Month 12 Generic drug name.
Spontaneous bleedings Month 1 - Month 3 - Month 6 - Month 12 Number from previous visit
Post-traumatic bleedings - localisation Month 1 - Month 3 - Month 6 - Month 12 Localisation of post-traumatic bleedings: joints, muscles, subcutaneous/ submucosal, abdominal/ gastrointestinal, retroperitoneal, genitourinary, CNS, pharyngeal, other.
Surgery/ Invasive Procedures: major Month 1 - Month 3 - Month 6 - Month 12 Number from previous visit.
Hemophilia Joint Health Score Day 0 - Month 6 - Month 12 Hemophilia Joint Health Score 2.1 for knee, elbow, ankle (left, right): score.
Target Joints Day 0 - Month 6 - Month 12 Target joints: knee, elbow, ankle, hip, shoulder, carpal, other (left, right): yes/ no.
Administration of efmorococtog alfa for correction of bleedings Month 1 - Month 3 - Month 6 - Month 12 Dose of efmorococtog alfa: IU.
Hospitalisations: duration Month 1 - Month 3 - Month 6 - Month 12 Total duration of all hospitalisations: days.
Administration of efmoroctocog alfa Day 0 - Month 1 - Month 3 - Month 6 - Month 12 Administration of efmoroctocog alfa, frequency: no. of injections per week.
Post-traumatic bleedings Month 1 - Month 3 - Month 6 - Month 12 Number from previous visit
Surgery/ Invasive Procedures: minor Month 1 - Month 3 - Month 6 - Month 12 Number from previous visit.
Ultrasound Scoring Day 0 - Month 6 - Month 12 Ultrasound scoring (HEAD-US): score.
Factor VIII Inhibitor Activity Day 0 - Month 1 - Month 3 - Month 6 - Month 12 Factor VIII Inhibitor Activity: Nijmegen-Bethesda unit/mL.
Administration of efmoroctocog alfa: factor consumption Day 0 - Month 1 - Month 3 - Month 6 - Month 12 Administration of efmoroctocog alfa, factor consumption: IU per week, IU/kg per week (calculation).
Trial Locations
- Locations (1)
League of Clinical Research (LeagueCRR)
🇷🇺Moscow, Russian Federation