Hemophilia A
Conditions
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.
Interventions
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
Sponsors
Bayer
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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 | At least 4 years | The maximum intensity of each safety event should be assigned to one of the following categories: mild, moderate or severe |
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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) | At least 4 years | 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. |
| Number of patients with abnormal findings as assessed by neurological examination | At least 4 years | — |
Countries
Austria, Germany, Greece, Italy, Slovenia, Spain
Outcome results
None listed