The goal of this study is to give gather more information on how safe and well Jivi works in patients with severe hemophilia A. Jivi has been approved by various regulatory agencies, including the FDA, Health Canada, Japanese Health Authority and the European Medicinal Agency. 25 patients will be enrolled and will stay for 1 to 2 years in this study depending on their treatment frequency. Researcher will monitor during the course of the study whether patients are developing antibodies (a protein made by the body in response to the drug) affecting the effectiveness of Jivi. In addition information on bleedings and patient's wellbeing will be collected.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
32
The recommended starting dose is every 5 days treatment (45 IU/kg)- An assessment of response to treatment will be performed at the next scheduled visit after 10-15 ED (8-10 weeks). Participants may be assigned to different dosing regimens (every 7 days or 2x/week) or continue with every 5 days regimen, according to individual bleeding tendency and needs at investigator's discretion.
UMHAT Tsaritsa Joanna-ISUL EAD Sofia
Sofia, Bulgaria
MHAT Sveta Marina EAD
Varna, Bulgaria
Aarhus Universitetshospital, Skejby
Arhus N, Denmark
LAIKO General Hospital of Athens
Athens, Greece
A.O. Pugliese-Ciaccio
Catanzaro, Calabria, Italy
A.O.U. Policlinico Umberto I
Rome, Lazio, Italy
Fondazione Policlinico Universitario Agostino Gemelli IRCCS
Rome, Lazio, Italy
Oslo Universitetssykehus HF, Rikshospitalet
Oslo, Norway
Uniwersyteckie Centrum Kliniczne
Gdansk, Poland
SP Szpital Kliniczny Nr 1
Wroclaw, Poland
...and 3 more locations
FVIII Inhibitor Development by the Nijmegen Bethesda Assay
FVIII inhibitor testing was performed using the Nijmegen-modified Bethesda assay. A positive inhibitor result was defined as a threshold of ≥0.6 BU/mL at the central laboratory and had to be confirmed with a second blood sample. After confirmation of the positive result, the inhibitor was to be reported as a serious adverse event (SAE).
Time frame: Observed for 100 exposure days (EDs), up to 2 years
Number of Participants With Treatment-emergent Adverse Events (TEAEs)
Treatment-emergent AEs were defined as those that started after the first dose of study drug and up to 7 days after the last dose.
Time frame: Observed for 100 exposure days (EDs), up to 2 years
Development of Treatment-emergent Anti-PEG Antibodies
Anti-PEG antibody: antibody against the PEG moiety determined by enzyme-linked immunosorbent assay (ELISA). For participants with a positive result, IgM antibodies were tested.
Time frame: Observed for 100 exposure days (EDs), up to 2 years
Annualized Bleeding Rate (ABR)
ABR is number of all bleeds per individual treatment period annualized to a 1-year time interval.
Time frame: Observed for 100 exposure days (EDs), up to 2 years
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