Clinical trial NCT04461639: 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
Official title: Observational Study Evaluating Long-term Safety of Real-world Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hemophilia A
Lead sponsor: Bayer
Sponsor class: INDUSTRY
First posted: 2020-07-08
Enrollment: 62
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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

Brief summary: 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.


Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with safety events
timeFrame: At least 4 years
measure: Duration of safety events
timeFrame: At least 4 years
measure: Number of participants with safety events leading to a change of treatment
timeFrame: At least 4 years
measure: Number of participants with safety events per intensity
timeFrame: At least 4 years
measure: Number of participants with safety events with outcome of death
timeFrame: At least 4 years
measure: Number of participants with safety events related to inhibitor development
timeFrame: At least 4 years
Secondary outcomes:
measure: Number of adverse reactions (ARs) that are defined within the system organ classes nervous system and psychiatric disorders
timeFrame: At least 4 years
measure: Number of adverse reactions (ARs) related to hepatic or renal function
timeFrame: At least 4 years
measure: Change from baseline in creatinine
timeFrame: At least 4 years
measure: Change from baseline in estimated glomerular filtration rate (eGFR)
timeFrame: At least 4 years
measure: Change from baseline in alanine transaminase (ALT)
timeFrame: At least 4 years
measure: Change from baseline in aspartate aminotransferase (AST)
timeFrame: At least 4 years
measure: Change from baseline in bilirubin
timeFrame: At least 4 years
measure: Testing for PEG plasma levels (baseline and end of study)
timeFrame: At least 4 years
measure: Number of patients with abnormal findings as assessed by neurological examination
timeFrame: At least 4 years
Other outcomes:
Locations:
city: Multiple Locations
country: Austria
facility: Many Locations
city: Multiple Locations
country: Germany
facility: Many Locations
city: Multiple Locations
country: Greece
facility: Many Locations
city: Multiple Locations
country: Italy
facility: Many Locations
city: Multiple Locations
country: Slovenia
facility: Many Locations
city: Multiple Locations
country: Spain
facility: Many Locations
Study officials:
References: