Brief Title
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
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
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
Health-related quality of life
Secondary Outcome
Spontaneous bleedings
Condition
Hemophilia A
Intervention
Efmoroctocog Alfa Injection [Eloctate]
Study Arms / Comparison Groups
Patients receiving Efmoroctocog alfa
Description: 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.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
30
Start Date
February 15, 2021
Completion Date
November 15, 2022
Primary Completion Date
August 15, 2022
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.
Gender
All
Ages
12 Years - 25 Years
Accepts Healthy Volunteers
No
Contacts
, 0079166950530, [email protected]
Location Countries
Russian Federation
Location Countries
Russian Federation
Administrative Informations
NCT ID
NCT04728217
Organization ID
RELOQ
Responsible Party
Sponsor
Study Sponsor
The League of Clinical Research, Russia
Study Sponsor
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Verification Date
January 2021