Observational Study Evaluating Long-term Safety of Real-world Treatment With Damoctocog Alfa Pegol in Previously Treated Patients With Hemophilia A
Trial Snapshot
- Phase
- Not Applicable
- Status
- Active, not recruiting
- Sponsor
- Enrollment
- 62
- Locations
- 6
- Primary Endpoint
- Number of participants with safety events with outcome of death
Study Overview
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.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 12 Years to — (Child, Adult, Older Adult)
- Sex
- All
- Accepts 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
Arms & Interventions
Damoctocog alfa pegol
Participants with hemophilia A received damoctocog alfa pegol as prophylaxis treatment prescribed by the physician as part of normal clinical practice.
Intervention: Damoctocog alfa pegol (Jivi, BAY94-9027) (Drug)
Outcomes
Primary Outcomes
Number of participants with safety events with outcome of death
Time Frame: At least 4 years
Number of participants with safety events related to inhibitor development
Time Frame: At least 4 years
Duration of safety events
Time Frame: At least 4 years
Number of participants with safety events
Time Frame: At least 4 years
Number of participants with safety events leading to a change of treatment
Time Frame: At least 4 years
Number of participants with safety events per intensity
Time Frame: At least 4 years
The maximum intensity of each safety event should be assigned to one of the following categories: mild, moderate or severe
Secondary Outcomes
- Change from baseline in aspartate aminotransferase (AST)(At least 4 years)
- Number of adverse reactions (ARs) related to hepatic or renal function(At least 4 years)
- Testing for PEG plasma levels (baseline and end of study)(At least 4 years)
- Change from baseline in bilirubin(At least 4 years)
- Number of patients with abnormal findings as assessed by neurological examination(At least 4 years)
- Change from baseline in creatinine(At least 4 years)
- Change from baseline in alanine transaminase (ALT)(At least 4 years)
- Number of adverse reactions (ARs) that are defined within the system organ classes nervous system and psychiatric disorders(At least 4 years)
- Change from baseline in estimated glomerular filtration rate (eGFR)(At least 4 years)