A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France

NCT03655340 | Completed

• 1 other identifier: Sobi.Alprolix-001
• Study Type: observational
• Target: 91
• Locations: 1 country
• Sites: 18

Brief Summary

Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.

Conditions

Haemophilia B

Keywords

Blood Coagulation Disorder; Hematologic Diseases; Coagulation Protein Disorder; Hemorrhagic Disorder; Genetic Diseases, Inborn

Outcome Measures

Primary Outcomes (5)

• Annualised bleeding rate (ABR) (prophylactic treatment)

  Assessed by diary

  24 months

• Annualised injection frequency (prophylactic treatment)

  Assessed by diary

  24 months

• Annualised factor consumption (International Unit [IU]) (prophylactic treatment)

  Assessed by diary

  24 months

• Amount of factor Product used to treat a bleed (on-demand treatment)

  Assessed by diary

  24 months

• Number of injections to treat a bleed (on-demand treatment)

  Assessed by diary

  24 months

Study Arms (2)

Prophylactic patients

Alprolix will be prescribed according to local practice and administered by patients with haemophilia B for prophylactic treatment

Drug: Alprolix

On demand patients

Alprolix will be prescribed according to local practice and administered by patients with haemophilia B for on-demand treatment

Drug: Alprolix

Interventions

Alprolix DRUG

Extended half-life factor IX product

Also known as: Eftrenonacog alfa, rFIXFc

On demand patients; Prophylactic patients

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

All eligible patients who present for a routine Clinical visit will be asked to participate in the study by the treating physician at participating haemophilia treatment centres in France.

You may qualify if:

• Have a diagnosis of haemophilia B and been treated previously with factor IX Product
• Have started Alprolix treatment prior to enrolment visit, or at enrolment prescribed treatment with Alprolix, regardless of participation in the study
• Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations

You may not qualify if:

• Participation in an investigational medicinal product trial at enrolment visit

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead

Study Sites (18)

Swedish Orphan Biovitrum Research Site (CHU de Bordeaux)

Bordeaux, France

Location

Swedish Orphan Biovitrum Research Site

Brest, France

Location

Swedish Orphan Biovitrum Research Site

Caen, France

Location

Swedish Orphan Biovitrum Research Site

Chambéry, France

Location

Swedish Orphan Biovitrum Research Site

Clermont-Ferrand, France

Location

Swedish Orphan Biovitrum Research Site

Dijon, France

Location

Swedish Orphan Biovitrum Research Site

La Réunion, France

Location

Swedish Orphan Biovitrum Research Site

Lille, France

Location

Swedish Orphan Biovitrum Research Site

Marseille, France

Location

Swedish Orphan Biovitrum Research Site

Montpellier, France

Location

Swedish Orphan Biovitrum Research Site

Nantes, France

Location

Swedish Orphan Biovitrum Research Site

Nîmes, France

Location

Swedish Orphan Biovitrum research site

Paris, France

Location

Swedish Orphan Biovitrum research site

Poitiers, France

Location

Swedish Orphan Biovitrum research site

Rennes, France

Location

Swedish Orphan Biovitrum Research Site

Rouen, France

Location

Swedish Orphan Biovitrum research site

Strasbourg, France

Location

Swedish Orphan Biovitrum Research Site

Toulouse, France

Location

Related Publications (1)

• Chambost H, Repesse Y, Genre-Volot F, Desprez D, Castet SM, Vanderbecken S, Zidi M, Gandossi C, Nuesch E, Palmborg H, Santagostino E; B-SURE Study Group. Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France. Ther Adv Hematol. 2025 Jan 26;16:20406207241311535. doi: 10.1177/20406207241311535. eCollection 2025.

  PMID: 39872008 DERIVED

MeSH Terms

Conditions

Hemophilia B; Blood Coagulation Disorders; Hematologic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn

Interventions

factor IX Fc fusion protein

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Officials

• Elena Santagostino, MD

  Swedish Orphan Biovitrum

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: August 30, 2018
• First Posted: August 31, 2018
• Study Start: September 12, 2018
• Primary Completion: March 16, 2022
• Study Completion: March 16, 2022
• Last Updated: September 19, 2024

Record last verified: 2024-09

Locations

FR (18)