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.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

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
Are there results?: Not yet: the study is under way

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.

City, postcode or hospital

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

Other trials in France

About this study

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.

3 Dec 2021Dec 2027

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)

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

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia

All Haemophilia B trials

Study at a glance

From the registry record, last updated by the sponsor on 8 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Observatory of Patients With Haemophilia B Treated by IdElvion®
ClinicalTrials.gov ID: NCT05086575
Acronym / sponsor ID: OrPHEe / OrPHEe study
Conditions: Haemophilia B
Keywords: FIX, Coagulation FIX
Study type: Observational
Recruitment status: Active, not recruiting
Enrolment: 222 participants (actual)
Lead sponsor: CSL BehringIndustry (pharma, biotech, devices)
Study start: 3 Dec 2021 (actual)
Primary completion: Dec 2027 (estimated)
Study completion: Dec 2027 (estimated)
First posted: 21 Oct 2021 (actual)
Results: Not posted in the source record

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Study design

Observational model: Cohort
Time perspective: Prospective

Who may qualify

Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult
Study population: Haemophilia B patients treated with Idelvion®

Key inclusion criteria (3 listed)

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

Key exclusion criteria (3 listed)

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

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

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
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

8 secondary outcomes

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

Results and safety

This study is still under way. Results are not available in the source record yet.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 8 Jan 2025.

Related trials for Haemophilia B

Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
Not yet recruitingPhase 1
Lentiviral FIX Gene Therapy
Hemophilia B
RecruitingPhase 3
A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
Hemophilia

Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Hasan Catovic study director

CSL Behring SA

28 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

An observational study of Haemophilia B, run by CSL Behring at 28 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 8 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 28 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

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). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05086575.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Jan 2025.