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 (REAL)

• ClinicalTrials.gov Identifier: NCT05395858
• Recruitment Status: Terminated
• First Posted: May 27, 2022
• Last Update Posted: November 28, 2023
• Sponsor: Bayer
• Information provided by (Responsible Party): Bayer

Tracking Information

• First Submitted Date: April 28, 2022
• First Posted Date: May 27, 2022
• Last Update Posted Date: November 28, 2023
• Actual Study Start Date: January 17, 2023
• Actual Primary Completion Date: November 6, 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 25, 2022)

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

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: May 25, 2022)

• 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 ]
  Only applicable for patients who discontinue damoctocog alfa pegol treatment regime.
• 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 ]

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: 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

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.

• Detailed Description: Not Provided
• Study Type: Observational
• Study Design: Observational Model: Cohort; Time Perspective: Retrospective
• Target Follow-Up Duration: Not Provided
• Biospecimen: Not Provided
• 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.
• Condition: Hemophilia A

Intervention

Other: No intervention

Retrospective analysis using database without any intervention assigned in the study.

Study Groups/Cohorts

Hemophilia A patients

Patients will be identified via standard medical charts or electronic medical records (EMRs).

Intervention: Other: No intervention

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Terminated
• Actual Enrollment (submitted: November 22, 2023): 14
• Original Estimated Enrollment (submitted: May 25, 2022): 20
• Actual Study Completion Date: November 6, 2023
• Actual Primary Completion Date: November 6, 2023 (Final data collection date for primary outcome measure)

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 12 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT05395858
• Other Study ID Numbers: 21280
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

Plan Description

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.

• Current Responsible Party: Bayer
• Original Responsible Party: Same as current
• Current Study Sponsor: Bayer
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Bayer
• Verification Date: November 2023