Clinical trial NCT04728217: Health Related Quality of Life of Youth and Young Adults With Haemophilia A
Official title: Health Related Quality of Life of Youth and Young Adults With Haemophilia A Treated With Efmoroctocog Alfa in Russia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Hemophilia A
Keywords: Haemophilia A
Health-related Quality of Life
Efmoroctocog alfa
Lead sponsor: The League of Clinical Research, Russia
Sponsor class: OTHER
First posted: 2021-01-28
Enrollment: 30
Sex: ALL
Minimum age (years): 12
Maximum age (years): 25
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: A 12-month prospective open-label, single-arm multicentre study.
Primary purpose: PREVENTION

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

Eligibility criteria: 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.

Primary outcomes:
measure: Health-related quality of life
timeFrame: Month 12
Secondary outcomes:
measure: Spontaneous bleedings
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Spontaneous bleedings - localisation
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Post-traumatic bleedings
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Post-traumatic bleedings - localisation
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Administration of efmorococtog alfa for correction of bleedings
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Surgery/ Invasive Procedures: major
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Surgery/ Invasive Procedures: minor
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Administration of efmorococtog alfa for correction of bleedings for Surgery/ Invasive Procedures
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Hospitalisations
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Hospitalisations: duration
timeFrame: Month 1 - Month 3 - Month 6 - Month 12
measure: Hemophilia Joint Health Score
timeFrame: Day 0 - Month 6 - Month 12
measure: Target Joints
timeFrame: Day 0 - Month 6 - Month 12
measure: Ultrasound Scoring
timeFrame: Day 0 - Month 6 - Month 12
measure: Factor VIII Activity
timeFrame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
measure: Factor VIII Inhibitor Activity
timeFrame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
measure: Administration of efmoroctocog alfa
timeFrame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
measure: Administration of efmoroctocog alfa: factor consumption
timeFrame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
measure: Previous treatment/ prophylaxis of haemophilia
timeFrame: Day 0 - Month 1 - Month 3 - Month 6 - Month 12
Other outcomes:
Locations:
city: Moscow
country: Russian Federation
facility: League of Clinical Research (LeagueCRR)
Study officials:
References: