Observatory of Patients With Haemophilia B Treated by IdElvion®

Haemophilia B Clinical Trial

— OrPHEe  

Official title:

Observatory of Patients With Haemophilia B Treated by IdElvion®

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05086575
• Other study ID #: OrPHEe study
• Secondary ID: 2021-A00468-33
• Status: Recruiting
• Start date: December 3, 2021
• Est. completion date: December 2027

Study information

• Verified date: April 2024
• Source: CSL Behring
• Contact: Diane Bracquart
• Phone: 610-878-4000
• Email: diane.bracquart[@]cslbehring.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: December 2027
• Est. primary completion date: December 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Haemophilia B
• Hemophilia A
• Hemophilia B

Locations

Country	Name	City	State
France	CHU Amiens - Picardie	Amiens
France	CH Annecy Genevois	Annecy
France	CHRU Besançon	Besançon
France	CHU Bordeaux - Hôpital Pellegrin	Bordeaux
France	CHRU Brest	Brest
France	CHU Caen	Caen
France	CHU Clermont-Ferrand	Clermont-Ferrand
France	CHU Dijon	Dijon
France	Hôpital Simone Veil	Eaubonne
France	CHU de Grenoble	Grenoble
France	CRC-MHC Hôpital Kremlin Bicêtre	Le Kremlin-Bicêtre
France	CH du Mans	Le Mans
France	CHRU Lille	Lille
France	CHU de Limoges	Limoges
France	Hôpital Edouard Herriot	Lyon
France	CHU Montpellier	Montpellier
France	CHRU Nancy	Nancy
France	CHU Nantes	Nantes
France	CHU Nice	Nice
France	Hôpital Necker Enfants Malades	Paris
France	CHU de Reims	Reims
France	CHU Rennes	Rennes
France	CHU Rouen	Rouen
France	CHU Saint-Etienne	Saint-Étienne
France	CHRU Strasbourg	Strasbourg
France	CHU Toulouse	Toulouse
France	CHU Tours	Tours
France	Hôpital André Mignot - CH Versailles	Versailles

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annual bleeding rates (overall, spontaneous and traumatic) under the scope of long-term prophylaxis or on-demand treatment of non-surgical bleeding events		Up to 36 months
Primary	Number of spontaneous bleeding events per patient		Up to 36 months
Primary	Number of Idelvion® infusions and doses injected by infusion required to prevent and resolve non-surgical bleeding episodes		Up to 36 months
Primary	Incidence of non-surgical bleeding episodes		Up to 36 months
Secondary	The number of infusions and total dose of Idelvion® (in IU/kg) required to prevent or treat surgical bleeding episodes		up to 36 months
Secondary	Incidence of surgical bleeding episodes		up to 36 months
Secondary	The number of infusions and total dose of Idelvion® (in IU/kg) required to cover a high risk of bleeding		up to 36 months
Secondary	The number of infusions and total dose of Factor IX consumed in the year prior to taking Idelvion®		up to 36 months
Secondary	The number of infusions and total dose of Idelvion®		up to 36 months
Secondary	The type and incidence of adverse events (AE) in particular severe AE,and AE related to Idelvion®		up to 36 months
Secondary	Completion of the following questionnaire: EQ-5D-3L for adults		up to 36 months
Secondary	Completion of the following questionnaire: EQ-5D-Y for children from 8 to 12 years		up to 36 months