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

NCT ID: NCT04953000

Last Updated: 2023-07-11

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

94 participants

Study Classification

OBSERVATIONAL

Study Start Date

2021-08-19

Study Completion Date

2023-04-04

Brief Summary

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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

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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

Conditions

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Hemophilia A

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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All Participants

Male participants with severe or moderate hemophilia A who have been treated with FVIII concentrate octocog alfa (Advate) during at least 12 months prior to the study enrollment, who started octocog alfa treatment in 2021 or currently being treated with octocog alfa will be observed in this study.

No interventions assigned to this group

Eligibility Criteria

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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.
Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Takeda

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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City Children's Hospital No. 1

Kazan', Tatarstan Republic, Russia

Site Status

Chelyabinsk Regional Children's Clinical Hospital

Chelyabinsk, , Russia

Site Status

Children's Regional Clinical Hospital

Khabarovsk, , Russia

Site Status

Krasnoyarsk Regional Clinical Center for Maternal and Child Health

Krasnoyarsk, , Russia

Site Status

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

Moscow, , Russia

Site Status

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

Nal'chik, , Russia

Site Status

State Novosibirsk Regional Clinical Hospital

Novosibirsk, , Russia

Site Status

Regional Children's Clinical Hospital

Rostov-on-Don, , Russia

Site Status

City Polyclinic No. 37

Saint Petersburg, , Russia

Site Status

Regional Children's Clinical Hospital No. 1

Vladivostok, , Russia

Site Status

Volgograd Regional Clinical Oncological Dispensary

Volgograd, , Russia

Site Status

Voronezh Regional Children's Clinical Hospital No. 1

Voronezh, , Russia

Site Status

State Autonomous Healthcare Institution of the Sverdlovsk Region "Regional Children's Clinical Hospital"

Yekaterinburg, , Russia

Site Status

Countries

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Russia

Related Links

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https://clinicaltrials.takeda.com/study-detail/60e84d641f1122001e30a94c

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

Other Identifiers

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MACS-2020-051901

Identifier Type: OTHER

Identifier Source: secondary_id

TAK-761-4001

Identifier Type: -

Identifier Source: org_study_id

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