Haemophilia BOrPHEeNCT05086575
Observatory of Patients With Haemophilia B Treated by IdElvion®
An observational study of Haemophilia B, run by CSL Behring at 28 sites in France.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia B
- How many people
- 222 participants
- Where
- 28 sitesFrance
- Study dates
- 3 Dec 2021to Dec 2027
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Haemophilia B patients treated with Idelvion®
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
28 sites in 1 country.
28 sites in 1 country
France28 sites
CHU Amiens - Picardie
Amiens
CH Annecy Genevois
Annecy
CHRU Besançon
Besançon
CHU Bordeaux - Hôpital Pellegrin
Bordeaux
CHRU Brest
Brest
CHU Caen
Caen
CHU Clermont-Ferrand
Clermont-Ferrand
CHU Dijon
Dijon
Hôpital Simone Veil
Eaubonne
CHU de Grenoble
Grenoble
CRC-MHC Hôpital Kremlin Bicêtre
Le Kremlin-Bicêtre
CH du Mans
Le Mans
CHRU Lille
Lille
CHU de Limoges
Limoges
Hôpital Edouard Herriot
Lyon
CHU Montpellier
Montpellier
CHRU Nancy
Nancy
CHU Nantes
Nantes
CHU Nice
Nice
Hôpital Necker Enfants Malades
Paris
CHU de Reims
Reims
CHU Rennes
Rennes
CHU Rouen
Rouen
CHU Saint-Etienne
Saint-Etienne
CHRU Strasbourg
Strasbourg
CHU Toulouse
Toulouse, 31059
CHU Tours
Tours
Hôpital André Mignot - CH Versailles
Versailles
About this study
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.
Read the 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.
- Conditions
- Haemophilia B
- Keywords
- FIX, Coagulation FIX
- Medical subject headings
- Hemophilia B
What is being measured
The primary outcome is the main question the study is designed to answer.
Annual bleeding rates (overall, spontaneous and traumatic) under the scope of long-term prophylaxis or on-demand treatment of non-surgical bleeding events
Measured: Up to 36 months
Number of spontaneous bleeding events per patient
Measured: Up to 36 months
Number of Idelvion® infusions and doses injected by infusion required to prevent and resolve non-surgical bleeding episodes
Measured: Up to 36 months
Incidence of non-surgical bleeding episodes
Measured: Up to 36 months
Secondary outcomes (8)
The number of infusions and total dose of Idelvion® (in IU/kg) required to prevent or treat surgical bleeding episodes
Measured: up to 36 months
Incidence of surgical bleeding episodes
Measured: up to 36 months
The number of infusions and total dose of Idelvion® (in IU/kg) required to cover a high risk of bleeding
Measured: up to 36 months
The number of infusions and total dose of Factor IX consumed in the year prior to taking Idelvion®
Measured: up to 36 months
The number of infusions and total dose of Idelvion®
Measured: up to 36 months
The type and incidence of adverse events (AE) in particular severe AE,and AE related to Idelvion®
Measured: up to 36 months
Completion of the following questionnaire: EQ-5D-3L for adults
Measured: up to 36 months
Completion of the following questionnaire: EQ-5D-Y for children from 8 to 12 years
Measured: up to 36 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2021
- Study start3 Dec 2021
- Primary completionDec 2027 (estimated)
- Study completionDec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia BStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 222 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hasan Catovic, Study director, CSL Behring SA
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05086575
- Sponsor's protocol ID
- OrPHEe study
- Other IDs
- 2021-A00468-33
- Acronym
- OrPHEe
- Status last verified
- Jan 2025
- First submitted
- 28 Sep 2021
- Last updated
- 8 Jan 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No