Observatory of Patients With Haemophilia B Treated by IdElvion® (OrPHEe)
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| ClinicalTrials.gov Identifier: NCT05086575 |
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Recruitment Status :
Recruiting
First Posted : October 21, 2021
Last Update Posted : March 7, 2023
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| Condition or disease |
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| Haemophilia B |
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 |
| 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 |
- 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 ]
- Number of spontaneous bleeding events per patient [ Time Frame: Up to 36 months ]
- Number of Idelvion® infusions and doses injected by infusion required to prevent and resolve non-surgical bleeding episodes [ Time Frame: Up to 36 months ]
- Incidence of non-surgical bleeding episodes [ Time Frame: Up to 36 months ]
- 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 ]
- Incidence of surgical bleeding episodes [ Time Frame: up to 36 months ]
- 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 ]
- The number of infusions and total dose of Factor IX consumed in the year prior to taking Idelvion® [ Time Frame: up to 36 months ]
- The number of infusions and total dose of Idelvion® [ Time Frame: up to 36 months ]
- The type and incidence of adverse events (AE) in particular severe AE,and AE related to Idelvion® [ Time Frame: up to 36 months ]
- Completion of the following questionnaire: EQ-5D-3L for adults [ Time Frame: up to 36 months ]
- Completion of the following questionnaire: EQ-5D-Y for children from 8 to 12 years [ Time Frame: up to 36 months ]
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.
| Ages Eligible for Study: | Child, Adult, Older Adult |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Probability Sample |
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
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
| Contact: Diane Bracquart | 610-878-4000 | diane.bracquart@cslbehring.com |
Show 25 study locations
| Study Director: | Hasan Catovic | CSL Behring SA |
| 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 |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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FIX, Coagulation FIX |
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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 |