Study to Learn More About the Safety of Drug Jivi Over a Long Period of Time in Previously Treated Patients With Hemophilia A (Bleeding Disorder Resulting From a Lack of FVIII) Who Are Receiving Jivi Regularly at Their Treating Doctors to Prevent Bleeding (HA-SAFE)

Observational Study Evaluating Long-term Safety of Real-world Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A

In this observational study researchers want to learn more about the safety of drug Jivi over a long period of time. Jivi (generic name: Damoctocog alfa pegol) is an approved blood clotting Factor VIII (FVIII) medication for the treatment of hemophilia A (bleeding disorder resulting from a lack of FVIII). It is manufactured via recombinant technology and has an extended half-live, i.e. it will stay longer in the body than other FVIII products. Therefore Jivi acts longer in the body which reduces the frequency of drug injections. This study will enroll previously treated patients with hemophilia A who are receiving Jivi regularly at their treating doctors to prevent bleeding. Observation for each patient will last for at least 4 years, and medical data will be collected during patients' routine visits at their treating doctors.

Study Overview

• Status: Active, not recruiting
• Conditions: Hemophilia A
• Intervention / Treatment: Drug: Damoctocog alfa pegol (Jivi, BAY94-9027)

• Study Type: Observational
• Enrollment (Actual): 62

Contacts and Locations

Study Locations

• Austria
  • Multiple Locations, Austria
    • Many Locations
• Germany
  • Multiple Locations, Germany
    • Many Locations
• Greece
  • Multiple Locations, Greece
    • Many Locations
• Italy
  • Multiple Locations, Italy
    • Many Locations
• Slovenia
  • Multiple Locations, Slovenia
    • Many Locations
• Spain
  • Multiple Locations, Spain
    • Many Locations

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

All previously treated patients (PTPs) with hemophilia A receiving prophylaxis with damoctocog alfa pegol will be eligible to be enrolled in the study. Indications and contra-indications according to the local market authorization will be carefully considered

Description

Inclusion Criteria:

• Signed informed consent/assent will be obtained before any study-related activities
• PTPs with hemophilia A assigned to Jivi prophylaxis treatment
• Negative FVIII inhibitor test before study entry
• Decision to initiate treatment with commercially available Jivi has been made by the treating physician before and independently from the decision to include the patient in this study

Exclusion Criteria:

• Known or suspected contraindications to Jivi or related products
• Mental incapacity, unwillingness or other barriers precluding adequate understanding or cooperation
• Participation in an investigational program with interventions outside of routine clinical practice

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Damoctocog alfa pegol; Participants with hemophilia A received damoctocog alfa pegol as prophylaxis treatment prescribed by the physician as part of normal clinical practice.	Drug: Damoctocog alfa pegol (Jivi, BAY94-9027); Different prophylaxis regimens with damoctocog alfa pegol following approved local labels or any other regimen prescribed by the physician as part of normal clinical practice

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of participants with safety events		At least 4 years
Duration of safety events		At least 4 years
Number of participants with safety events leading to a change of treatment		At least 4 years
Number of participants with safety events per intensity	The maximum intensity of each safety event should be assigned to one of the following categories: mild, moderate or severe	At least 4 years
Number of participants with safety events with outcome of death		At least 4 years
Number of participants with safety events related to inhibitor development		At least 4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adverse reactions (ARs) that are defined within the system organ classes nervous system and psychiatric disorders		At least 4 years
Number of adverse reactions (ARs) related to hepatic or renal function		At least 4 years
Change from baseline in creatinine		At least 4 years
Change from baseline in estimated glomerular filtration rate (eGFR)		At least 4 years
Change from baseline in alanine transaminase (ALT)		At least 4 years
Change from baseline in aspartate aminotransferase (AST)		At least 4 years
Change from baseline in bilirubin		At least 4 years
Testing for PEG plasma levels (baseline and end of study)	PEG (Polyethylene Glycol)-plasma levels at baseline and end of study will be analyzed only if PEG-plasma levels were collected in local routine clinical practice at the investigator's discretion.	At least 4 years
Number of patients with abnormal findings as assessed by neurological examination		At least 4 years

Collaborators and Investigators

• Sponsor: Bayer

Study record dates

Study Major Dates

• Study Start (Actual): May 14, 2021
• Primary Completion (Estimated): June 30, 2028
• Study Completion (Estimated): June 30, 2028

Study Registration Dates

• First Submitted: June 18, 2020
• First Submitted That Met QC Criteria: July 2, 2020
• First Posted (Actual): July 8, 2020

Study Record Updates

• Last Update Posted (Actual): April 12, 2024
• Last Update Submitted That Met QC Criteria: April 11, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII
• Other Study ID Numbers: 20904

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

IPD 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.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No