A Study of Standard and Individualized Prophylactic Treatment With Advate in Men and Boys With Severe Hemophilia A

Hemophilia A Clinical Trial

— HAPKIDO  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04953000
• Other study ID #: TAK-761-4001
• Secondary ID: MACS-2020-051901
• Status: Completed
• Start date: August 19, 2021
• Est. completion date: April 4, 2023

Study information

• Verified date: July 2023
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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 information / eligibility

• Status: Completed
• Enrollment: 94
• Est. completion date: April 4, 2023
• Est. primary completion date: April 4, 2023
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A

Locations

Country	Name	City	State
Russian Federation	Chelyabinsk Regional Children's Clinical Hospital	Chelyabinsk
Russian Federation	City Children's Hospital No. 1	Kazan	Tatarstan
Russian Federation	Children's Regional Clinical Hospital	Khabarovsk
Russian Federation	Krasnoyarsk Regional Clinical Center for Maternal and Child Health	Krasnoyarsk
Russian Federation	Morozovskaya Children's City Clinical Hospital of the Department of Healthcare of the city of Moscow	Moscow
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	State Novosibirsk Regional Clinical Hospital	Novosibirsk
Russian Federation	Regional Children's Clinical Hospital	Rostov-on-Don
Russian Federation	City Polyclinic No. 37	St. Petersburg
Russian Federation	Regional Children's Clinical Hospital No. 1	Vladivostok
Russian Federation	Volgograd Regional Clinical Oncological Dispensary	Volgograd
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

Sponsors (1)

Lead Sponsor	Collaborator
Takeda

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time per Week Spent With Factor VIII (FVIII) Trough Level Below 1 Percent (%) on the Standard Prophylaxis and After Pharmacokinetic (PK) -tailored Prophylaxis	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.	From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary	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	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.	From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary	Percentage of Participants With Upgraded Dosing Regimen Based on PK-assessment	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.	Baseline up to Visit 3 (Month 12)
Secondary	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	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.	From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary	Percentage of Participants With FVIII Trough Level Permanently Above 1% on Standard Prophylaxis vs on PK-tailored Prophylaxis	Percentage of participants with FVIII trough level permanently above 1% on standard and PK-tailored prophylaxis will be reported.	From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary	Correlation Between the Time per Week Spent With FVIII Trough Level Below 1% and Spontaneous Annualized Bleeding Rate (ABR) on Standard Prophylaxis	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.	From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary	Correlation Between the Time per Week Spent With FVIII Trough Level Below 1% and Spontaneous Annualized Joint Bleeding Rate (AJBR) on Standard Prophylaxis	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.	From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary	Total Consumption of FVIII Before and After PK-tailored Prophylaxis in Participants With Upgraded Dosing Regimen	Total consumption of FVIII before and after PK-tailored prophylaxis in participants with upgraded dosing regimen will be reported.	From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary	Percentage of Participants With Positive Dynamics of Body Status After Transfer From Standard to Individualized Prophylaxis	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.	Baseline up to Visit 3 (Month 12)
Secondary	Percentage of Participants Categorized Based on Number of Hospitalizations due to Insufficient Bleeding Control Before and After PK-tailored Prophylaxis	Percentage of participants categorized based on number of hospitalization due to insufficient bleeding control before and after PK-tailored Prophylaxis will be reported.	From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary	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	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.	From 12 months prior to study enrollment up to Visit 3 (Month 12)

External Links

•   To obtain more information on the study, click here/on this link