A Study of Standard and Individualized Prophylactic Treatment With Advate in Men and Boys With Severe Hemophilia A
- Conditions
- Hemophilia A
- Registration Number
- NCT04953000
- Lead Sponsor
- Takeda
- 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
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- Male
- Target Recruitment
- 94
-
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.
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Time per Week Spent With Factor VIII (FVIII) Trough Level Below 1 Percent (%) on the Standard Prophylaxis and After Pharmacokinetic (PK) -tailored Prophylaxis From 12 months prior to study enrollment up to Visit 3 (Month 12) PK-tailoring prophylaxis is defined as FVIII replacement prophylactic therapy based on PK parameters of each particular participant. Time per week spent with Through FVIII level below 1% on standard and PK-tailored prophylaxis will be reported.
- Secondary Outcome Measures
Name Time Method Correlation Between the Time per Week Spent With FVIII Trough Level Below 1% and Spontaneous Annualized Joint Bleeding Rate (AJBR) on Standard Prophylaxis From 12 months prior to study enrollment up to Visit 3 (Month 12) Correlation between the time per week spent with FVIII trough level below 1% and spontaneous AJBR on standard prophylaxis will be assessed using Pearson's correlation coefficient.
Total Consumption of FVIII Before and After PK-tailored Prophylaxis in Participants With Upgraded Dosing Regimen From 12 months prior to study enrollment up to Visit 3 (Month 12) Total consumption of FVIII before and after PK-tailored prophylaxis in participants with upgraded dosing regimen will be reported.
Percentage of Participants With Positive Dynamics of Body Status After Transfer From Standard to Individualized Prophylaxis Baseline up to Visit 3 (Month 12) Positive dynamics of body status is related with increased physical activity (change in body mass index \[BMI\], waist: hip ratio \[WHR\]. Percentage of participants with positive dynamics of body status after transfer from standard to individualized prophylaxis will be reported.
Percentage of Participants With FVIII Trough Level Permanently Above 1% on Standard Prophylaxis vs on PK-tailored Prophylaxis From 12 months prior to study enrollment up to Visit 3 (Month 12) Percentage of participants with FVIII trough level permanently above 1% on standard and PK-tailored prophylaxis will be reported.
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 From 12 months prior to study enrollment up to Visit 3 (Month 12) Percentage of participants with more than 19 hours per week with FVIII trough level below 1% on standard and PK-tailored prophylaxis will be reported.
Percentage of Participants Categorized Based on Number of Hospitalizations due to Insufficient Bleeding Control Before and After PK-tailored Prophylaxis From 12 months prior to study enrollment up to Visit 3 (Month 12) Percentage of participants categorized based on number of hospitalization due to insufficient bleeding control before and after PK-tailored Prophylaxis will be reported.
Percentage of Participants Categorized Based on Number of Days Away From Work and School/Institute Due to Insufficient Bleeding Control Before and After PK-tailored Prophylaxis From 12 months prior to study enrollment up to Visit 3 (Month 12) Percentage of participants categorized based on number of days away from work and school/institute due to insufficient bleeding control before and after PK-tailored prophylaxis will be reported.
Percentage of Participants With Upgraded Dosing Regimen Based on PK-assessment Baseline up to Visit 3 (Month 12) Upgraded dosing regimen includes dose upgrade, dosing intervals modification, and/or FVIII trough level upgrade to above 1%. Percentage of participants with upgraded dosing regimen based on PK-assessment will be reported.
Percentage of Time per Week Spent With FVIII Trough Level Below 1%, 1 to less than (<) 3%, and Greater Than or Equal to (>=) 3% While on Standard and Individualized Prophylaxis From 12 months prior to study enrollment up to Visit 3 (Month 12) Individualized prophylaxis is defined as prophylactic therapy based on PK parameters of each individual participant. Percentage of time per week spent with FVIII through level below 1%, 1- \<3%, and \>=3% while on standard and individualized prophylaxis will be reported.
Correlation Between the Time per Week Spent With FVIII Trough Level Below 1% and Spontaneous Annualized Bleeding Rate (ABR) on Standard Prophylaxis From 12 months prior to study enrollment up to Visit 3 (Month 12) Correlation between the time per week spent with FVIII trough level below 1% and spontaneous ABR on standard prophylaxis will be assessed using Pearson's correlation coefficient.
Trial Locations
- Locations (13)
Children's Regional Clinical Hospital
🇷🇺Khabarovsk, Russian Federation
State Novosibirsk Regional Clinical Hospital
🇷🇺Novosibirsk, Russian Federation
City Polyclinic No. 37
🇷🇺St. Petersburg, Russian Federation
Regional Children's Clinical Hospital No. 1
🇷🇺Vladivostok, Russian Federation
Voronezh Regional Children's Clinical Hospital No. 1
🇷🇺Voronezh, Russian Federation
State Autonomous Healthcare Institution of the Sverdlovsk Region "Regional Children's Clinical Hospital"
🇷🇺Yekaterinburg, Russian Federation
Krasnoyarsk Regional Clinical Center for Maternal and Child Health
🇷🇺Krasnoyarsk, Russian Federation
Chelyabinsk Regional Children's Clinical Hospital
🇷🇺Chelyabinsk, Russian Federation
Volgograd Regional Clinical Oncological Dispensary
🇷🇺Volgograd, Russian Federation
City Children's Hospital No. 1
🇷🇺Kazan, Tatarstan, Russian Federation
Regional Children's Clinical Hospital
🇷🇺Rostov-on-Don, Russian Federation
State Budgetary Institution of Healthcare "Republican children's clinical hospital" of the Ministry of Health of the Kabardino-Balkar Republic
🇷🇺Nalchik, Russian Federation
Morozovskaya Children's City Clinical Hospital of the Department of Healthcare of the city of Moscow
🇷🇺Moscow, Russian Federation