Observatory of Patients With Haemophilia B Treated by IdElvion® (OrPHEe)

CSL Behring

Status: Active, not recruiting

Conditions: Haemophilia B

Study type: Observational

Funder types: Industry

Identifiers

NCT05086575

2021-A00468-33 (Other Identifier)

OrPHEe study

Details and patient eligibility

About

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.

Full 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.

Enrollment: 222 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Have agreed to participate in the observatory after receiving written information on the purpose of the study and the personal data to be collected (agreement of parents for under-age patients);
• Present with haemophilia B and are currently being treated or have previously been treated with Idelvion® as a long-term prophylactic treatment, on-demand treatment or short-term treatment for surgical procedures or to cover periods with a high-risk of bleeding.
• Do not have FIX targeted antibodies at the time of the inclusion visit

Exclusion criteria

• Refusal by the patient or his/her legal representative to participate in the study;
• Existence of a contraindication to the use of Idelvion® treatment (known hypersensitivity to FIX or hamster proteins);
• Simultaneous participation in an interventional clinical study on a drug