Brief Title
A Study of Standard and Individualized Prophylactic Treatment With Advate in Men and Boys With Severe Hemophilia A
Official Title
Local, Multicenter, Non-interventional, Ambispective Study in Severe Hemophilia A Patients on Standard and PK-tailored Prophylaxis With Octocog Alfa (Advate®) in the Russian Federation
Brief Summary
The main aim of this study is to learn about changes in the lowest blood levels of Factor VIII in men and boys when upgraded from standard prophylaxis with Advate to individualized prophylaxis with Advate. No study medicines will be provided to participants in this study. The study sponsor will not be involved in how participants are treated but will provide instructions on how the clinics will record what happens during the study. Participants will need to visit the study doctor 3 times in total during the study. During these visits, study data will be collected by the study doctor.
Detailed Description
Local, Multicenter, Non-interventional, Ambispective Study in Severe Hemophilia A Patients on Standard and PK-tailored Prophylaxis With Octocog Alfa (Advate®) in the Russian Federation
Study Type
Observational
Primary Outcome
Time per Week Spent With Factor VIII (FVIII) Trough Level Below 1 Percent (%) on the Standard Prophylaxis and After Pharmacokinetic (PK) -tailored Prophylaxis
Secondary Outcome
Percentage of Participants With More Than 19 Hours per Week Spent With FVIII Trough Level Below 1% on Standard Prophylaxis Versus on PK-tailored Prophylaxis
Condition
Hemophilia A
Study Arms / Comparison Groups
All Participants
Description: Male participants with severe or moderate hemophilia A who have been treated with FVIII concentrate octocog alfa (Advate) during at least 12 months prior to the study enrollment, who started octocog alfa treatment in 2021 or currently being treated with octocog alfa will be observed in this study.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
94
Start Date
August 19, 2021
Completion Date
February 10, 2023
Primary Completion Date
February 10, 2023
Eligibility Criteria
Inclusion Criteria: - Male participants of all ages with severe hemophilia A (FVIII ˂1%) or moderate hemophilia A with severe bleeding phenotype who: - had been on standard prophylaxis or PK-tailored prophylaxis with octocog alfa during at least 12 months prior to the study enrollment (150 participants) or who started Advate® treatment in 2021 (20 participants), - is being treated with octocog alfa at the moment of enrolment with any of the above mentioned treatment modalities, and - had been assigned octocog alfa provision in Federal reimbursement program 2021 - Availability of participants' records sufficient for data collection according to the study objectives during the retrospective period of the study for participants who had been on standard prophylaxis or PK-tailored prophylaxis with octocog alfa during at least 12 months prior to the study enrollment - Written informed consent provided by the participant or, in case of children below 14 years of age, by participant's parent or participant's legally acceptable representative. Exclusion Criteria: - Failure to obtain the participant's written informed consent - Participation in any interventional study of products for hemophilia or other hemostasis disturbances treatment during 12 months prior to the study enrollment for participants who has retrospective data collection period and during 12 months after the study enrollment for all participants.
Gender
Male
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Study Director, ,
Location Countries
Russian Federation
Location Countries
Russian Federation
Administrative Informations
NCT ID
NCT04953000
Organization ID
TAK-761-4001
Secondary IDs
MACS-2020-051901
Responsible Party
Sponsor
Study Sponsor
Takeda
Study Sponsor
Study Director, Study Director, Takeda
Verification Date
September 2022