Clinical trial NCT04953000: 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
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2021-07-07
Enrollment: 94
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: PROBABILITY_SAMPLE
Study population: Male participants of all ages being treated with octocog alfa (Advate) for severe hemophilia A in 2021 will be enrolled in this study.

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

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.

Primary outcomes:
measure: Time per Week Spent With Factor VIII (FVIII) Trough Level Below 1 Percent (%) on the Standard Prophylaxis and After Pharmacokinetic (PK) -tailored Prophylaxis
timeFrame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary outcomes:
measure: 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
timeFrame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
measure: Percentage of Participants With Upgraded Dosing Regimen Based on PK-assessment
timeFrame: Baseline up to Visit 3 (Month 12)
measure: 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
timeFrame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
measure: Percentage of Participants With FVIII Trough Level Permanently Above 1% on Standard Prophylaxis vs on PK-tailored Prophylaxis
timeFrame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
measure: Correlation Between the Time per Week Spent With FVIII Trough Level Below 1% and Spontaneous Annualized Bleeding Rate (ABR) on Standard Prophylaxis
timeFrame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
measure: Correlation Between the Time per Week Spent With FVIII Trough Level Below 1% and Spontaneous Annualized Joint Bleeding Rate (AJBR) on Standard Prophylaxis
timeFrame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
measure: Total Consumption of FVIII Before and After PK-tailored Prophylaxis in Participants With Upgraded Dosing Regimen
timeFrame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
measure: Percentage of Participants With Positive Dynamics of Body Status After Transfer From Standard to Individualized Prophylaxis
timeFrame: Baseline up to Visit 3 (Month 12)
measure: Percentage of Participants Categorized Based on Number of Hospitalizations due to Insufficient Bleeding Control Before and After PK-tailored Prophylaxis
timeFrame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
measure: 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
timeFrame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
Other outcomes:
Locations:
city: Kazan
country: Russian Federation
facility: City Children's Hospital No. 1
state: Tatarstan
city: Chelyabinsk
country: Russian Federation
facility: Chelyabinsk Regional Children's Clinical Hospital
city: Khabarovsk
country: Russian Federation
facility: Children's Regional Clinical Hospital
city: Krasnoyarsk
country: Russian Federation
facility: Krasnoyarsk Regional Clinical Center for Maternal and Child Health
city: Moscow
country: Russian Federation
facility: Morozovskaya Children's City Clinical Hospital of the Department of Healthcare of the city of Moscow
city: Nalchik
country: Russian Federation
facility: State Budgetary Institution of Healthcare "Republican children's clinical hospital" of the Ministry of Health of the Kabardino-Balkar Republic
city: Novosibirsk
country: Russian Federation
facility: State Novosibirsk Regional Clinical Hospital
city: Rostov-on-Don
country: Russian Federation
facility: Regional Children's Clinical Hospital
city: St. Petersburg
country: Russian Federation
facility: City Polyclinic No. 37
city: Vladivostok
country: Russian Federation
facility: Regional Children's Clinical Hospital No. 1
city: Volgograd
country: Russian Federation
facility: Volgograd Regional Clinical Oncological Dispensary
city: Voronezh
country: Russian Federation
facility: Voronezh Regional Children's Clinical Hospital No. 1
city: Yekaterinburg
country: Russian Federation
facility: State Autonomous Healthcare Institution of the Sverdlovsk Region "Regional Children's Clinical Hospital"
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: