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

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.

Study Overview

• Status: Active, not recruiting
• Conditions: Haemophilia B

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.

• Study Type: Observational
• Enrollment (Actual): 222

Contacts and Locations

Study Locations

• France
  • Amiens, France
    • Chu Amiens - Picardie
  • Annecy, France
    • CH Annecy Genevois
  • Besançon, France
    • CHRU Besançon
  • Bordeaux, France
    • CHU bordeaux - Hopital Pellegrin
  • Brest, France
    • CHRU BREST
  • Caen, France
    • CHU Caen
  • Clermont-Ferrand, France
    • Chu Clermont-Ferrand
  • Dijon, France
    • CHU Dijon
  • Eaubonne, France
    • Hôpital Simone Veil
  • Grenoble, France
    • CHU de Grenoble
  • Le Kremlin-Bicêtre, France
    • CRC-MHC Hôpital Kremlin Bicêtre
  • Le Mans, France
    • CH du Mans
  • Lille, France
    • CHRU LILLE
  • Limoges, France
    • CHU de Limoges
  • Lyon, France
    • Hôpital Edouard Herriot
  • Montpellier, France
    • CHU Montpellier
  • Nancy, France
    • CHRU Nancy
  • Nantes, France
    • CHU Nantes
  • Nice, France
    • CHU Nice
  • Paris, France
    • Hopital Necker Enfants Malades
  • Reims, France
    • CHU de Reims
  • Rennes, France
    • CHU Rennes
  • Rouen, France
    • CHU Rouen
  • Saint-Étienne, France
    • CHU Saint-Etienne
  • Strasbourg, France
    • CHRU Strasbourg
  • Toulouse, France, 31059
    • CHU Toulouse
  • Tours, France
    • CHU Tours
  • Versailles, France
    • Hôpital André Mignot - CH Versailles

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Probability Sample

Study Population

Haemophilia B patients treated with Idelvion®

Description

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

Study Plan

How is the study designed?

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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
Number of spontaneous bleeding events per patient	Up to 36 months
Number of Idelvion® infusions and doses injected by infusion required to prevent and resolve non-surgical bleeding episodes	Up to 36 months
Incidence of non-surgical bleeding episodes	Up to 36 months

Secondary Outcome Measures

Outcome Measure	Time Frame
The number of infusions and total dose of Idelvion® (in IU/kg) required to prevent or treat surgical bleeding episodes	up to 36 months
Incidence of surgical bleeding episodes	up to 36 months
The number of infusions and total dose of Idelvion® (in IU/kg) required to cover a high risk of bleeding	up to 36 months
The number of infusions and total dose of Factor IX consumed in the year prior to taking Idelvion®	up to 36 months
The number of infusions and total dose of Idelvion®	up to 36 months
The type and incidence of adverse events (AE) in particular severe AE,and AE related to Idelvion®	up to 36 months
Completion of the following questionnaire: EQ-5D-3L for adults	up to 36 months
Completion of the following questionnaire: EQ-5D-Y for children from 8 to 12 years	up to 36 months

Collaborators and Investigators

• Sponsor: CSL Behring
• Investigators: Study Director: Hasan Catovic, CSL Behring SA

Study record dates

Study Major Dates

• Study Start (Actual): December 3, 2021
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2027

Study Registration Dates

• First Submitted: September 28, 2021
• First Submitted That Met QC Criteria: October 7, 2021
• First Posted (Actual): October 21, 2021

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 7, 2025
• Last Verified: January 1, 2025

More Information

Terms related to this study

• Keywords: FIX, Coagulation FIX
• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Genetic Diseases, X-Linked; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemophilia A; Hemophilia B
• Other Study ID Numbers: OrPHEe study; 2021-A00468-33 (Other Identifier: ANSM)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No