We're building a better ClinicalTrials.gov. Check it out and tell us what you think!
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT05086575
Recruitment Status : Recruiting
First Posted : October 21, 2021
Last Update Posted : March 7, 2023
Information provided by (Responsible Party):
CSL Behring

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.

Condition or disease
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.

Layout table for study information
Study Type : Observational
Estimated Enrollment : 100 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Observatory of Patients With Haemophilia B Treated by IdElvion®
Actual Study Start Date : December 3, 2021
Estimated Primary Completion Date : September 2026
Estimated Study Completion Date : September 2026

Resource links provided by the National Library of Medicine

Drug Information available for: Idelvion

Primary Outcome Measures :
  1. Annual bleeding rates (overall, spontaneous and traumatic) under the scope of long-term prophylaxis or on-demand treatment of non-surgical bleeding events [ Time Frame: Up to 36 months ]
  2. Number of spontaneous bleeding events per patient [ Time Frame: Up to 36 months ]
  3. Number of Idelvion® infusions and doses injected by infusion required to prevent and resolve non-surgical bleeding episodes [ Time Frame: Up to 36 months ]
  4. Incidence of non-surgical bleeding episodes [ Time Frame: Up to 36 months ]

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

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
Haemophilia B patients treated with Idelvion®

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05086575

Layout table for location contacts
Contact: Diane Bracquart 610-878-4000 diane.bracquart@cslbehring.com

Show Show 25 study locations
Sponsors and Collaborators
CSL Behring
Layout table for investigator information
Study Director: Hasan Catovic CSL Behring SA
Layout table for additonal information
Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT05086575    
Other Study ID Numbers: OrPHEe study
2021-A00468-33 ( Other Identifier: ANSM )
First Posted: October 21, 2021    Key Record Dates
Last Update Posted: March 7, 2023
Last Verified: March 2023

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by CSL Behring:
FIX, Coagulation FIX
Additional relevant MeSH terms:
Layout table for MeSH terms
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked