Clinical trial NCT05395858: 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
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
Study type: OBSERVATIONAL
Overall status: TERMINATED
Conditions: Hemophilia A
Keywords: Bleeding disorder
Coagulation factor VIII
Blood Coagulation Disorders
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2022-05-27
Enrollment: 14
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

Brief 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. 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.


Eligibility criteria: Inclusion Criteria:

* 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
* 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.

Exclusion Criteria:

* Diagnosis of any other bleeding/coagulation disorder other than hemophilia A.
* Participation in any past or current damoctocog alfa pegol interventional trial.

Primary outcomes:
measure: Frequency of damoctocog alfa pegol infusions
timeFrame: From index date through the date of central IRB approval (3 years and 8 months)
Secondary outcomes:
measure: Descriptive summary of pre-damoctocog alfa pegol treatment regimen
timeFrame: 6 months prior to the index date.
measure: Descriptive summary of damoctocog alfa pegol treatment regimen.
timeFrame: Up to 5 years from index date
measure: Descriptive summary of post-damoctocog alfa pegol treatment regimen, if applicable.
timeFrame: Up to 4 years
measure: Descriptive summary of changes in pre-damoctocog alfa pegol treatment regimes
timeFrame: 6 months prior to the index date.
measure: Descriptive summary for reasons for treatment discontinuation of pre-damoctocog alfa pegol treatment regimes.
timeFrame: 6 months prior to the index date.
measure: Annualized number of spontaneous, joint, and trauma bleeds before initiation of treatment with damoctocog alfa pegol.
timeFrame: 6 months prior to the index date.
measure: Annualized number of spontaneous, joint, and trauma bleeds post initiation of treatment with damoctocog alfa pegol.
timeFrame: Up to 5 years from index date
Other outcomes:
Locations:
city: Sacramento
country: United States
facility: University of California, Davis
state: California
city: New Orleans
country: United States
facility: Tulane University
state: Louisiana
city: Minneapolis
country: United States
facility: Regents of the University of Minnesota
state: Minnesota
Study officials:
References: