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Completed

NCT Number: NCT04953000

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

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.

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Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Sex eligibility

Male

Study type

Observational

Primary location

City Children's Hospital No. 1, Kazan', Tatarstan Republic, Russia

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About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

  1. Time per Week Spent With Factor VIII (FVIII) Trough Level Below 1 Percent (%) on the Standard Prophylaxis and After Pharmacokinetic (PK) -tailored Prophylaxis

    Time frame: 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 outcomes

  1. 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

    Time frame: 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.

  2. Percentage of Participants With Upgraded Dosing Regimen Based on PK-assessment

    Time frame: 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.

  3. 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

    Time frame: 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.

  4. Percentage of Participants With FVIII Trough Level Permanently Above 1% on Standard Prophylaxis vs on PK-tailored Prophylaxis

    Time frame: 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.

  5. Correlation Between the Time per Week Spent With FVIII Trough Level Below 1% and Spontaneous Annualized Bleeding Rate (ABR) on Standard Prophylaxis

    Time frame: 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.

  6. Correlation Between the Time per Week Spent With FVIII Trough Level Below 1% and Spontaneous Annualized Joint Bleeding Rate (AJBR) on Standard Prophylaxis

    Time frame: 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.

  7. Total Consumption of FVIII Before and After PK-tailored Prophylaxis in Participants With Upgraded Dosing Regimen

    Time frame: 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.

  8. Percentage of Participants With Positive Dynamics of Body Status After Transfer From Standard to Individualized Prophylaxis

    Time frame: 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.

  9. Percentage of Participants Categorized Based on Number of Hospitalizations due to Insufficient Bleeding Control Before and After PK-tailored Prophylaxis

    Time frame: 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.

  10. 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

    Time frame: 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.

Sponsors and collaborators

Lead sponsor

Takeda

Industry

Registry information

Official study 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

Acronym: HAPKIDO

Important dates

Study start
2021
Primary completion
2023
Study completion
2023
First posted
Jul 7, 2021
Registry last updated
Jul 11, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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