Clinical trial NCT05086575: Observatory of Patients With Haemophilia B Treated by IdElvion®
Official title: Observatory of Patients With Haemophilia B Treated by IdElvion®
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Haemophilia B
Keywords: FIX, Coagulation FIX
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2021-10-21
Enrollment: 222
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Haemophilia B patients treated with Idelvion®

Brief 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.

Eligibility criteria: 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

Primary outcomes:
measure: Annual bleeding rates (overall, spontaneous and traumatic) under the scope of long-term prophylaxis or on-demand treatment of non-surgical bleeding events
timeFrame: Up to 36 months
measure: Number of spontaneous bleeding events per patient
timeFrame: Up to 36 months
measure: Number of Idelvion® infusions and doses injected by infusion required to prevent and resolve non-surgical bleeding episodes
timeFrame: Up to 36 months
measure: Incidence of non-surgical bleeding episodes
timeFrame: Up to 36 months
Secondary outcomes:
measure: The number of infusions and total dose of Idelvion® (in IU/kg) required to prevent or treat surgical bleeding episodes
timeFrame: up to 36 months
measure: Incidence of surgical bleeding episodes
timeFrame: up to 36 months
measure: The number of infusions and total dose of Idelvion® (in IU/kg) required to cover a high risk of bleeding
timeFrame: up to 36 months
measure: The number of infusions and total dose of Factor IX consumed in the year prior to taking Idelvion®
timeFrame: up to 36 months
measure: The number of infusions and total dose of Idelvion®
timeFrame: up to 36 months
measure: The type and incidence of adverse events (AE) in particular severe AE,and AE related to Idelvion®
timeFrame: up to 36 months
measure: Completion of the following questionnaire: EQ-5D-3L for adults
timeFrame: up to 36 months
measure: Completion of the following questionnaire: EQ-5D-Y for children from 8 to 12 years
timeFrame: up to 36 months
Other outcomes:
Locations:
city: Amiens
country: France
facility: CHU Amiens - Picardie
city: Annecy
country: France
facility: CH Annecy Genevois
city: Besançon
country: France
facility: CHRU Besançon
city: Bordeaux
country: France
facility: CHU Bordeaux - Hôpital Pellegrin
city: Brest
country: France
facility: CHRU Brest
city: Caen
country: France
facility: CHU Caen
city: Clermont-Ferrand
country: France
facility: CHU Clermont-Ferrand
city: Dijon
country: France
facility: CHU Dijon
city: Eaubonne
country: France
facility: Hôpital Simone Veil
city: Grenoble
country: France
facility: CHU de Grenoble
city: Le Kremlin-Bicêtre
country: France
facility: CRC-MHC Hôpital Kremlin Bicêtre
city: Le Mans
country: France
facility: CH du Mans
city: Lille
country: France
facility: CHRU Lille
city: Limoges
country: France
facility: CHU de Limoges
city: Lyon
country: France
facility: Hôpital Edouard Herriot
city: Montpellier
country: France
facility: CHU Montpellier
city: Nancy
country: France
facility: CHRU Nancy
city: Nantes
country: France
facility: CHU Nantes
city: Nice
country: France
facility: CHU Nice
city: Paris
country: France
facility: Hôpital Necker Enfants Malades
city: Reims
country: France
facility: CHU de Reims
city: Rennes
country: France
facility: CHU Rennes
city: Rouen
country: France
facility: CHU Rouen
city: Saint-Étienne
country: France
facility: CHU Saint-Etienne
city: Strasbourg
country: France
facility: CHRU Strasbourg
city: Toulouse
country: France
facility: CHU Toulouse
city: Tours
country: France
facility: CHU Tours
city: Versailles
country: France
facility: Hôpital André Mignot - CH Versailles
Study officials:
affiliation: CSL Behring SA
name: Hasan Catovic
role: STUDY_DIRECTOR
References: