Hemophilia A
Conditions
Keywords
Haemophilia A, Health-related Quality of Life, Efmoroctocog alfa
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
Interventions
DRUGEfmoroctocog Alfa Injection [Eloctate]
Efmoroctocog alfa should be administered according to Summary of Product Characteristics (SmPC)/ Instruction on Medical Use approved by Russian Ministry of Health ЛП-006034 (13.01.2020).
Sponsors
The League of Clinical Research, Russia
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
PREVENTION
Masking
NONE
Intervention model description
A 12-month prospective open-label, single-arm multicentre study.
Eligibility
Sex/Gender
ALL
Age
12 Years to 25 Years
Healthy volunteers
No
Inclusion criteria
1. Signed consent form. 2. Adults, adolescents and children (12-25 years old) with haemophilia A with ABR ≥ 2 on at least one-year SHL FVIII prophylaxis.
Exclusion criteria
1. Patients who have had hypersensitivity reactions to efmoroctocog alfa or other constituents of the product. 2. History of Factor VIII inhibitors. 3. Patients who have other haemostatic disorders. 4. Patients participating in interventional studies.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| Post-traumatic 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. |
| Administration of efmorococtog alfa for correction of bleedings | Month 1 - Month 3 - Month 6 - Month 12 | Dose of efmorococtog alfa: IU. |
| Surgery/ Invasive Procedures: major | 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. |
| 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. |
| Spontaneous bleedings | 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. |
| Ultrasound Scoring | Day 0 - Month 6 - Month 12 | Ultrasound scoring (HEAD-US): score. |
| 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. |
| 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 | Day 0 - Month 1 - Month 3 - Month 6 - Month 12 | Administration of efmoroctocog alfa, frequency: no. of injections per week. |
| 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). |
| Previous treatment/ prophylaxis of haemophilia | Day 0 - Month 1 - Month 3 - Month 6 - Month 12 | Generic drug name. |
| Hospitalisations: duration | Month 1 - Month 3 - Month 6 - Month 12 | Total duration of all hospitalisations: days. |
Countries
Russia
Outcome results
None listed