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

Takeda94 enrolled

Overview

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.

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

Enrollment

Conditions

Hemophilia A

Eligibility

Sex: MALE

Medical Language ↔ Plain English

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.

Locations (13)

City Children's Hospital No. 1

Kazan', Tatarstan Republic, Russia

Chelyabinsk Regional Children's Clinical Hospital

Chelyabinsk, Russia

Children's Regional Clinical Hospital

Khabarovsk, Russia

Krasnoyarsk Regional Clinical Center for Maternal and Child Health

Krasnoyarsk, Russia

Morozovskaya Children's City Clinical Hospital of the Department of Healthcare of the city of Moscow

Moscow, Russia

State Budgetary Institution of Healthcare "Republican children's clinical hospital" of the Ministry of Health of the Kabardino-Balkar Republic

Nal'chik, Russia

State Novosibirsk Regional Clinical Hospital

Novosibirsk, Russia

Regional Children's Clinical Hospital

Rostov-on-Don, Russia

City Polyclinic No. 37

Saint Petersburg, Russia

Regional Children's Clinical Hospital No. 1

Vladivostok, Russia

...and 3 more locations

Outcomes

Primary Outcomes

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.