Hemophilia AREALNCT05395858
A Study to Learn More About Treatment With Damoctocog Alfa Pegol, How it is Used in Every Day Practice ("Real-World"), and How Satisfied People Who Receive Damoctocog Alfa Pegol Are in United States (US) Hemophilia Treatment Centers
An observational study of Hemophilia A, run by Bayer at 3 sites in United States.
Trial summary
People with hemophilia A do not have enough of a protein found naturally in the blood called "clotting factor 8", also known as FVIII. This protein helps the blood to clump together to prevent and stop bleeding. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Hemophilia A
- How many people
- 14 participants
- Where
- 3 sitesUnited States
- Study dates
- 17 Jan 2023to 6 Nov 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: United States patients aged ≥12 years diagnosed with hemophilia A (congenital FVIII deficiency) who switched from a previous FVIII replacement treatment to damoctocog alfa pegol. No investigational products will be administered in this study.
Who may be able to join
- Male or female with a diagnosis of hemophilia A
- Previously or currently treated with damoctocog alfa pegol for at least 6 months.
- Treated with damoctocog alfa pegol per the US approved label to include:
- Aged ≥12 years at the time of damoctocog alfa pegol treatment initiation
- Previously treated for hemophilia A with an FVIII replacement therapy other than damoctocog alfa pegol
- Treatment modality of on-demand, prophylactic, intermittent prophylaxis or a combination thereof
Show all 13Show fewer
- Have data in the medical record at the participating site as follows:
- For a minimum of 6 months prior to the damoctocog alfa pegol initiation date
- For a minimum of 6 months post-damoctocog alfa pegol initiation date AND the patient was receiving treatment with damoctocog alfa pegol during this 6-month follow-up period AND this data is dated prior to the central institutional review board (IRB) approval date for the study
- To include the FVIII replacement therapy most recently received prior to the initiation of damoctocog alfa pegol
- For patients in the prospective cohort
- Signed informed consent
- Current treatment with damoctocog alfa pegol or discontinued use of damoctocog alfa pegol within 3 months prior to the date of enrollment into the retrospective cohort.
Who cannot join
- Diagnosis of any other bleeding/coagulation disorder other than hemophilia A.
- Participation in any past or current damoctocog alfa pegol interventional trial.
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Regents of the University of Minnesota
Minneapolis, Minnesota, 55455
Tulane University
New Orleans, Louisiana, 70112
University of California, Davis
Sacramento, California, 95817
About this study
Official title: REAL: A Pre-/Post-Intervention Descriptive Analysis and Cross-Sectional Survey to Evaluate Real-World Treatment Outcomes, Consumption, and Satisfaction With Damoctocog Alfa Pegol in US Hemophilia Treatment Centers
People with hemophilia A do not have enough of a protein found naturally in the blood called "clotting factor 8", also known as FVIII. This protein helps the blood to clump together to prevent and stop bleeding. People with lower levels of FVIII or FVIII that does not work properly may bleed for a long time from minor wounds, bleed into their joints, or have internal bleeding.
The study treatment, Jivi (also called damoctocog alfa pegol), is already available as a treatment for people aged 12 years and older with hemophilia A, to help prevent bleeding, also known as "prophylactic" treatment. It works by replacing the missing FVIII, or the FVIII that does not work properly. It can also be used to stop bleeding that has already occurred and prior to surgery to prevent bleeding.
Read more
The main goal of this study is to learn how damoctocog alfa pegol is used in the "real world" as a treatment in the United States (US) and how well it works and what other treatments patients use while receiving damoctocog alfa pegol treatment. It will also determine how satisfied people are with the treatment. There will be no required visits with a study doctor in this study.
The study will include about 20 male or female patients in the US aged 12 years and over who have hemophilia A. All the patients in this study will have switched from their previous FVIII replacement treatment to damoctocog alfa pegol. While the patients are receiving damoctocog alfa pegol, they will complete a survey to say how they feel about the treatment. Their doctors will also record information about their treatment with damoctocog alfa pegol and how well it is working.
This study will collect information from the patients' medical records and surveys. They will use this information to find out more about treatment with damoctocog alfa pegol under "real world" conditions. They will look at:
- how often the patients receive damoctocog alfa pegol and how much they use
- what other treatments the patients received before receiving damoctocog alfa pegol, how they used it and how much they used
- how well damoctocog alfa pegol works at preventing bleeding, and how it compares to previous products used.
- Conditions
- Hemophilia A
- Being tested
- No intervention
- Keywords
- Bleeding disorder, Coagulation factor VIII, Blood Coagulation Disorders
- Medical subject headings
- Hemophilia A, Hemostatic Disorders, Blood Coagulation Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionOther
Retrospective analysis using database without any intervention assigned in the study.
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A patients | Patients will be identified via standard medical charts or electronic medical records (EMRs). Receives: Other: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Frequency of damoctocog alfa pegol infusions
Reported as infusions. IRB: institutional review board
Measured: From index date through the date of central IRB approval (3 years and 8 months)
Secondary outcomes (7)
Descriptive summary of pre-damoctocog alfa pegol treatment regimen
Measured: 6 months prior to the index date.
Descriptive summary of damoctocog alfa pegol treatment regimen.
Measured: Up to 5 years from index date
Descriptive summary of post-damoctocog alfa pegol treatment regimen, if applicable.
Only applicable for patients who discontinue damoctocog alfa pegol treatment regime.
Measured: Up to 4 years
Descriptive summary of changes in pre-damoctocog alfa pegol treatment regimes
Measured: 6 months prior to the index date.
Descriptive summary for reasons for treatment discontinuation of pre-damoctocog alfa pegol treatment regimes.
Measured: 6 months prior to the index date.
Annualized number of spontaneous, joint, and trauma bleeds before initiation of treatment with damoctocog alfa pegol.
Measured: 6 months prior to the index date.
Annualized number of spontaneous, joint, and trauma bleeds post initiation of treatment with damoctocog alfa pegol.
Measured: Up to 5 years from index date
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 May 2022
- Study start17 Jan 2023
- Primary completion6 Nov 2023
- Study completion6 Nov 2023
Why it stopped: Bayer business decision to close the REAL study based on the following: Target objective of enrolling at least 20 patients into the retrospective part failed; Not enough available data that would be a meaningful addition to the literature.
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 BayerThe 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
- Retrospective
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bayer
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05395858
- Sponsor's protocol ID
- 21280
- Acronym
- REAL
- Status last verified
- Nov 2023
- First submitted
- 28 Apr 2022
- Last updated
- 28 Nov 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.
Study at a glance
From the registry record, last updated by the sponsor on 28 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- REAL: A Pre-/Post-Intervention Descriptive Analysis and Cross-Sectional Survey to Evaluate Real-World Treatment Outcomes, Consumption, and Satisfaction With Damoctocog Alfa Pegol in US Hemophilia Treatment Centers
- ClinicalTrials.gov ID
- NCT05395858
- Acronym / sponsor ID
- REAL / 21280
- Conditions
- Hemophilia A
- Keywords
- Bleeding disorder, Coagulation factor VIII, Blood Coagulation Disorders
- Study type
- Observational
- Recruitment status
- TerminatedReason stopped: Bayer business decision to close the REAL study based on the following: Target objective of enrolling at least 20 patients into the retrospective part failed; Not enough available data that would be a meaningful addition to the literature.
- Enrolment
- 14 participants (actual)
- Lead sponsor
- BayerIndustry (pharma, biotech, devices)
- Study start
- 17 Jan 2023 (actual)
- Primary completion
- 6 Nov 2023 (actual)
- Study completion
- 6 Nov 2023 (actual)
- First posted
- 27 May 2022 (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.
People with hemophilia A do not have enough of a protein found naturally in the blood called "clotting factor 8", also known as FVIII. This protein helps the blood to clump together to prevent and stop bleeding. People with lower levels of FVIII or FVIII that does not work properly may bleed for a long time from minor wounds, bleed into their joints, or have internal bleeding.
The study treatment, Jivi (also called damoctocog alfa pegol), is already available as a treatment for people aged 12 years and older with hemophilia A, to help prevent bleeding, also known as "prophylactic" treatment. It works by replacing the missing FVIII, or the FVIII that does not work properly. It can also be used to stop bleeding that has already occurred and prior to surgery to prevent bleeding.
The main goal of this study is to learn how damoctocog alfa pegol is used in the "real world" as a treatment in the United States (US) and how well it works and what other treatments patients use while receiving damoctocog alfa pegol treatment. It will also determine how satisfied people are with the treatment. There will be no required visits with a study doctor in this study.
The study will include about 20 male or female patients in the US aged 12 years and over who have hemophilia A. All the patients in this study will have switched from their previous FVIII replacement treatment to damoctocog alfa pegol. While the patients are receiving damoctocog alfa pegol, they will complete a survey to say how they feel about the treatment. Their doctors will also record information about their treatment with damoctocog alfa pegol and how well it is working.
This study will collect information from the patients' medical records and surveys. They will use this information to find out more about treatment with damoctocog alfa pegol under "real world" conditions. They will look at:
- how often the patients receive damoctocog alfa pegol and how much they use
- what other treatments the patients received before receiving damoctocog alfa pegol, how they used it and how much they used
- how well damoctocog alfa pegol works at preventing bleeding, and how it compares to previous products used.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A patients | – | Other: No interventionPatients will be identified via standard medical charts or electronic medical records (EMRs). |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- United States patients aged ≥12 years diagnosed with hemophilia A (congenital FVIII deficiency) who switched from a previous FVIII replacement treatment to damoctocog alfa pegol. No investigational products will be administered in this study.
Key inclusion criteria (13 listed)
- Male or female with a diagnosis of hemophilia A
- Previously or currently treated with damoctocog alfa pegol for at least 6 months.
- Treated with damoctocog alfa pegol per the US approved label to include:
- Have data in the medical record at the participating site as follows:
- For patients in the prospective cohort
Key exclusion criteria (2 listed)
- Diagnosis of any other bleeding/coagulation disorder other than hemophilia A.
- Participation in any past or current damoctocog alfa pegol interventional trial.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionRetrospective analysis using database without any intervention assigned in the study. | Other | Hemophilia A patients |
Primary outcome
Frequency of damoctocog alfa pegol infusions
Time frame: From index date through the date of central IRB approval (3 years and 8 months)
Reported as infusions. IRB: institutional review board
7 secondary outcomes
Descriptive summary of pre-damoctocog alfa pegol treatment regimen
Time frame: 6 months prior to the index date.
Descriptive summary of damoctocog alfa pegol treatment regimen.
Time frame: Up to 5 years from index date
Descriptive summary of post-damoctocog alfa pegol treatment regimen, if applicable.
Time frame: Up to 4 years
Descriptive summary of changes in pre-damoctocog alfa pegol treatment regimes
Time frame: 6 months prior to the index date.
Descriptive summary for reasons for treatment discontinuation of pre-damoctocog alfa pegol treatment regimes.
Time frame: 6 months prior to the index date.
Annualized number of spontaneous, joint, and trauma bleeds before initiation of treatment with damoctocog alfa pegol.
Time frame: 6 months prior to the index date.
Annualized number of spontaneous, joint, and trauma bleeds post initiation of treatment with damoctocog alfa pegol.
Time frame: Up to 5 years from index date
Results and safety
The study was stopped early (reason given: Bayer business decision to close the REAL study based on the following: Target objective of enrolling at least 20 patients into the retrospective part failed; Not enough available data that would be a meaningful addition to the literature.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Nov 2023.
- Individual participant dataSharing planned: No. Availability of this study's data will later be determined according to Bayer's commitment to the EFPIA/PhRMA "Principles for responsible clinical trial data sharing". This pertains to scope, timepoint and process of data access. As such, Bayer commits to sharing upon request from qualified researchers patient-level clinical trial data, study-level clinical trial data, and protocols from clinical trials in patients for medicines and indications approved in the US and EU as necessary for conducting legitimate research. This applies to data on new medicines and indications that have been approved by the EU and US regulatory agencies on or after January 01, 2014. Interested researchers can use www.vivli.org to request access to anonymized patient-level data and supporting documents from clinical studies to conduct research. Information on the Bayer criteria for listing studies and other relevant information is provided in the member section of the portal.
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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.
The record lists no contact. Use the official record to reach the sponsor.
3 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 Bayer at 3 sites in United States.
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 13 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: stopped early, as of the sponsor's last update on 28 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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 was stopped early (reason given: Bayer business decision to close the REAL study based on the following: Target objective of enrolling at least 20 patients into the retrospective part failed; Not enough available data that would be a meaningful addition to the literature.). Results are not available in the source record.
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?
Frequency of damoctocog alfa pegol infusions (time frame: From index date through the date of central IRB approval (3 years and 8 months)). 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 28 Nov 2023.