Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice
Trial Snapshot
- Phase
- Not Applicable
- Status
- Completed
- Sponsor
- Enrollment
- 50
- Locations
- 26
- Primary Endpoint
- Number of AESIs including seriousness, severity, relationship to therapy, outcome, and treatment discontinuation
Study Overview
Brief Summary
The study addresses the safety, utilisation and effectiveness of Obizur in the treatment of bleeding episodes in real-life clinical practice in Europe and the United States.
Study Design
- Study Type
- Observational
- Observational Model
- Cohort
- Time Perspective
- Prospective
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- •Adult participant (or legal representative) is willing to provide informed consent
- •Participant is being treated or was treated (treatment initiation within 30 days) with Obizur in routine clinical practice
Exclusion Criteria
- •Participant has known anaphylactic reactions to the active substance, hamster protein or to any of the following excipients: Polysorbate 80; sodium chloride; calcium chloride dihydrate; sucrose; Tris Base; Tris HCl; Tri-sodium citrate dihydrate; sterilized water for injections
- •Participant has participated in a clinical study involving a medicinal product or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving a medicinal product or device at study entry
Outcomes
Primary Outcomes
Number of AESIs including seriousness, severity, relationship to therapy, outcome, and treatment discontinuation
Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Adverse Events of Special Interest (AESI) are as follows: hypersensitivity reactions, thromboembolic events and dose dispensing medication errors.
Number of thromboembolic events
Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Thromboembolic events include disseminated intravascular coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial infarction and stroke.
Number of AEs and SAEs including seriousness, severity and outcome
Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
AE - adverse event, SAE - serious adverse event.
Number of dose dispensing medication errors
Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Dose dispensing medication erros include miscalculation of dose while prescribing (calculation of correct dose based on the participant's weight) or administration of the incorrect dose.
Secondary Outcomes
- Obizur treatment regimen, as available(From first administration of Obizur up to 180 days after the last administration of Obizur.)
- Other medication administered for haemostatic control, as available(From first administration of Obizur up to 180 days after the last administration of Obizur.)
- Overall effectiveness assessment for resolution of bleeding(From first administration of Obizur up to 180 days after the last administration of Obizur.)
- Immunogenicity; newly recognized anti-pFVIII inhibitor or increase in titre of anti-pFVIII inhibitors and evolution of titre over time(From first administration of Obizur up to 180 days after the last administration of Obizur.)
- Dose per infusion administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur(From first administration of Obizur up to 180 days after the last administration of Obizur.)
- Time to achieve bleeding control, death or change in haemostatic treatment other than Obizur(From first administration of Obizur up to 180 days after the last administration of Obizur.)
- Number of infusions administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur(From first administration of Obizur up to 180 days after the last administration of Obizur.)