Evaluating the safety and effectiveness of Fibryga for congenital fibrinogen deficiency
Post-marketing Observational Study on the Safety and Efficacy of Fibryga in Congenital Fibrinogen Deficiency
This study is testing how safe and effective Fibryga is for people of all ages with congenital fibrinogen deficiency who need treatment for bleeding.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 25 (estimated) |
| Sex | All |
| Sponsor | Octapharma Industry-sponsored |
| Locations | 4 sites (Ostrava and 3 other locations) |
| Trial ID | NCT03793426 on ClinicalTrials.gov |
What this trial studies
This observational study aims to gather real-world evidence on the safety and efficacy of Fibryga in patients with congenital fibrinogen deficiency. It will document the administration of Fibryga during routine clinical use for treating both minor and major bleeding events. By collecting data from various clinical settings, the study seeks to enhance understanding of Fibryga's performance beyond controlled clinical trials, ultimately supporting its benefit-risk profile. The study includes patients of all ages who require on-demand treatment for bleeding events.
Who should consider this trial
Good fit: Ideal candidates are patients of any age with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia who need treatment for bleeding events.
Not a fit: Patients with bleeding disorders other than congenital fibrinogen deficiency or those with acquired fibrinogen deficiency may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the real-world effectiveness and safety of Fibryga, improving treatment options for patients with congenital fibrinogen deficiency.
How similar studies have performed: Other observational studies have successfully gathered real-world evidence for similar treatments, indicating that this approach is valuable and not entirely novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients of any age with a documented diagnosis of congenital afibrinogenemia or hypofibrinogenemia expected to require on-demand in-hospital treatment for BEs with Fibryga Exclusion Criteria: * Bleeding disorder other than congenital fibrinogen deficiency * Patients with acquired fibrinogen deficiency or dysfibrinogenemia * Suspicion of an anti-fibrinogen inhibitor as indicated by previous in vivo recovery, if available, of \<0.5 (mg/dL)/(mg/kg); there is currently no standard test for inhibitors * Participation in an interventional clinical study at the time of or within 4 weeks prior to enrolment
Where this trial is running
Ostrava and 3 other locations
- Fakultní nemocnice Ostrava — Ostrava, Czechia (Recruiting)
- Gerinnungszentrum rhein-ruhr — Duisburg, Germany (Recruiting)
- Medizinische Klinik 2 / Institut für Transfusionsmedizin Universitätsklinikum — Frankfurt, Germany (Recruiting)
- Gerinnungszentrum Hochtaunus — Hamburg, Germany (Recruiting)
Study contacts
- Study coordinator: Gita Pezeshki, PhD
- Email: gita.pezeshki@octapharma.com
- Phone: +12016041125
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.