NCT05086575

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.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
222

participants targeted

Target at P75+ for all trials

Timeline
19mo left

Started Dec 2021

Longer than P75 for all trials

Geographic Reach
1 country

28 active sites

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress74%
Dec 2021Dec 2027

First Submitted

Initial submission to the registry

September 28, 2021

Completed
23 days until next milestone

First Posted

Study publicly available on registry

October 21, 2021

Completed
1 month until next milestone

Study Start

First participant enrolled

December 3, 2021

Completed
6 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2027

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2027

Last Updated

January 8, 2025

Status Verified

January 1, 2025

Enrollment Period

6 years

First QC Date

September 28, 2021

Last Update Submit

January 7, 2025

Conditions

Haemophilia B

Keywords

FIX, Coagulation FIX

Outcome Measures

Primary Outcomes (4)

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

    Up to 36 months

  • Number of spontaneous bleeding events per patient

    Up to 36 months

  • Number of Idelvion® infusions and doses injected by infusion required to prevent and resolve non-surgical bleeding episodes

    Up to 36 months

  • Incidence of non-surgical bleeding episodes

    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

    up to 36 months

  • Incidence of surgical bleeding episodes

    up to 36 months

  • The number of infusions and total dose of Idelvion® (in IU/kg) required to cover a high risk of bleeding

    up to 36 months

  • The number of infusions and total dose of Factor IX consumed in the year prior to taking Idelvion®

    up to 36 months

  • The number of infusions and total dose of Idelvion®

    up to 36 months

  • +3 more secondary outcomes

Eligibility Criteria

Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodProbability Sample
Study Population

Haemophilia B patients treated with Idelvion®

You may qualify if:

  • 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.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (28)

CHU Amiens - Picardie

Amiens, France

Location

CH Annecy Genevois

Annecy, France

Location

CHRU Besançon

Besançon, France

Location

CHU Bordeaux - Hôpital Pellegrin

Bordeaux, France

Location

CHRU Brest

Brest, France

Location

CHU Caen

Caen, France

Location

CHU Clermont-Ferrand

Clermont-Ferrand, France

Location

CHU Dijon

Dijon, France

Location

Hôpital Simone Veil

Eaubonne, France

Location

CHU de Grenoble

Grenoble, France

Location

CRC-MHC Hôpital Kremlin Bicêtre

Le Kremlin-Bicêtre, France

Location

CH du Mans

Le Mans, France

Location

CHRU Lille

Lille, France

Location

CHU de Limoges

Limoges, France

Location

Hôpital Edouard Herriot

Lyon, France

Location

CHU Montpellier

Montpellier, France

Location

CHRU Nancy

Nancy, France

Location

CHU Nantes

Nantes, France

Location

CHU Nice

Nice, France

Location

Hôpital Necker Enfants Malades

Paris, France

Location

CHU de Reims

Reims, France

Location

CHU Rennes

Rennes, France

Location

CHU Rouen

Rouen, France

Location

CHU Saint-Etienne

Saint-Etienne, France

Location

CHRU Strasbourg

Strasbourg, France

Location

CHU Toulouse

Toulouse, 31059, France

Location

CHU Tours

Tours, France

Location

Hôpital André Mignot - CH Versailles

Versailles, France

Location

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, InheritedBlood Coagulation DisordersHematologic DiseasesHemic and Lymphatic DiseasesCoagulation Protein DisordersHemorrhagic DisordersGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-Linked

Study Officials

  • Hasan Catovic

    CSL Behring SA

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 28, 2021

First Posted

October 21, 2021

Study Start

December 3, 2021

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

December 1, 2027

Last Updated

January 8, 2025

Record last verified: 2025-01

Locations

FR(28)