NCT05086575

Observatory of Patients With Haemophilia B Treated by IdElvion®

• Status: Active Not Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 222 (actual)
• Sponsor: CSL Behring · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

OrPHEe is a non-interventional, prospective and national study which aim is to record real life data in haemophilia B French patients treated with Idelvion® to confirm the efficacy and safety of this product established in clinical development studies.

Detailed description

Haemophilia B is a congenital coagulation disorder caused by a deficiency or anomaly of coagulation factor IX (FIX). The severity of the haemophilia depends on the extent of the FIX deficiency with clinical manifestations differing depending on the location of the bleed. Treatment of this disease involves the administration of the deficient factor, i.e. FIX, to patients. Depending on the severity of the disease and context, a range of treatment regimens are available (long-term prophylactic treatment for the prevention of non-surgical bleeds, short-term prophylactic treatment for high-risk periods, treatment for the prevention of surgical bleeds or on-demand curative treatment). CSL Behring has developed a long-acting recombinant FIX, i.e. rIX-FP (Idelvion®), to extend the intervals between the administrations of treatment while also providing a therapeutic benefit. Data from clinical studies clearly confirm the therapeutic benefit of Idelvion® in adult and paediatric patients with haemophilia B previously receiving an on-demand treatment for bleeding episodes, for long-term prophylaxis, as well as in patients undergoing surgery.

Conditions

• Haemophilia B

Timeline

• Start date: 2021-12-03
• Primary completion: 2027-12-01
• Completion: 2027-12-01
• First posted: 2021-10-21
• Last updated: 2025-01-08

Locations

28 sites across 1 country: France