Hemophilia AHAPKIDONCT04953000
A Study of Standard and Individualized Prophylactic Treatment With Advate in Men and Boys With Severe Hemophilia A
An observational study of Hemophilia A, run by Takeda at 13 sites in Russia.
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. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Hemophilia A
- How many people
- 94 participants
- Where
- 13 sitesRussia
- Study dates
- 19 Aug 2021to 4 Apr 2023
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- Men only
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
13 sites in 1 country.
13 sites in 1 country
Russia13 sites
Chelyabinsk Regional Children's Clinical Hospital
Chelyabinsk, 454087
City Children's Hospital No. 1
Kazan', Tatarstan Republic, 420034
Children's Regional Clinical Hospital
Khabarovsk, 680003
Krasnoyarsk Regional Clinical Center for Maternal and Child Health
Krasnoyarsk, 660074
Morozovskaya Children's City Clinical Hospital of the Department of Healthcare of the city of Moscow
Moscow, 119049
State Budgetary Institution of Healthcare "Republican children's clinical hospital" of the Ministry of Health of the Kabardino-Balkar Republic
Nal'chik, 360032
State Novosibirsk Regional Clinical Hospital
Novosibirsk, 630087
Regional Children's Clinical Hospital
Rostov-on-Don, 344015
City Polyclinic No. 37
Saint Petersburg, 191186
Regional Children's Clinical Hospital No. 1
Vladivostok, 690002
Volgograd Regional Clinical Oncological Dispensary
Volgograd, 400138
Voronezh Regional Children's Clinical Hospital No. 1
Voronezh, 394024
State Autonomous Healthcare Institution of the Sverdlovsk Region "Regional Children's Clinical Hospital"
Yekaterinburg, 620149
About this study
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
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.
Read the 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
- Conditions
- Hemophilia A
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: From 12 months prior to study enrollment up to Visit 3 (Month 12)
Secondary outcomes (10)
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.
Measured: From 12 months prior to study enrollment up to Visit 3 (Month 12)
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.
Measured: Baseline up to Visit 3 (Month 12)
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.
Measured: 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 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.
Measured: 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 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.
Measured: 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 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.
Measured: 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
Total consumption of FVIII before and after PK-tailored prophylaxis in participants with upgraded dosing regimen will be reported.
Measured: From 12 months prior to study enrollment up to Visit 3 (Month 12)
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.
Measured: Baseline up to Visit 3 (Month 12)
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.
Measured: 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
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.
Measured: From 12 months prior to study enrollment up to Visit 3 (Month 12)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jul 2021
- Study start19 Aug 2021
- Primary completion4 Apr 2023
- Study completion4 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by TakedaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 94 participants (actual)
Sponsor and investigators
- Lead sponsor
- Takeda
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04953000
- Sponsor's protocol ID
- TAK-761-4001
- Other IDs
- MACS-2020-051901
- Acronym
- HAPKIDO
- Status last verified
- Jul 2023
- First submitted
- 6 Jul 2021
- Last updated
- 11 Jul 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 11 Jul 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04953000
- Acronym / sponsor ID
- HAPKIDO / TAK-761-4001
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 94 participants (actual)
- Lead sponsor
- TakedaIndustry (pharma, biotech, devices)
- Study start
- 19 Aug 2021 (actual)
- Primary completion
- 4 Apr 2023 (actual)
- Study completion
- 4 Apr 2023 (actual)
- First posted
- 7 Jul 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 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. |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (6 listed)
- Male participants of all ages with severe hemophilia A (FVIII ˂1%) or moderate hemophilia A with severe bleeding phenotype who:
- 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.
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
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.
10 secondary outcomes
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 Upgraded Dosing Regimen Based on PK-assessment
Time frame: Baseline up to Visit 3 (Month 12)
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)
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)
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 Annualized Joint Bleeding Rate (AJBR) on Standard Prophylaxis
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
Time frame: From 12 months prior to study enrollment up to Visit 3 (Month 12)
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)
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 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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Jul 2023.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Takeda
13 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Takeda at 13 sites in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 11 Jul 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 13 sites in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 11 Jul 2023.