Observatory of Patients With Haemophilia B Treated by IdElvion®

Brief Title

Observatory of Patients With Haemophilia B Treated by IdElvion®

Official Title

Observatory of Patients With Haemophilia B Treated by 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.

Study Type

Observational

Primary Outcome

Annual bleeding rates (overall, spontaneous and traumatic) under the scope of long-term prophylaxis or on-demand treatment of non-surgical bleeding events

Secondary Outcome

 The number of infusions and total dose of Idelvion® (in IU/kg) required to prevent or treat surgical bleeding episodes

Condition

Haemophilia B

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Estimated Enrollment

Start Date

December 3, 2021

Completion Date

September 2026

Primary Completion Date

September 2026

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

Gender

All

Ages

N/A - N/A

Accepts Healthy Volunteers

No

Contacts

Hasan Catovic, 610-878-4000, [email protected]

Location Countries

France

Location Countries

France

Administrative Informations

NCT ID

NCT05086575

Organization ID

OrPHEe study

Secondary IDs

2021-A00468-33

Responsible Party

Sponsor

Study Sponsor

CSL Behring

Study Sponsor

Hasan Catovic, Study Director, CSL Behring SA

Verification Date

March 2023