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

NCT03901755 | Completed

• 1 other identifier: Sobi.Alprolix-002
• Study Type: observational
• Target: 151
• Locations: 9 countries
• Sites: 29

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

Hemophilia B

Keywords

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

Outcome Measures

Primary Outcomes (3)

• Annualised bleeding rate (ABR)

  Bleeding episodes assessed according to local practice

  24 months

• Annualised injection frequency

  Assessed by prescription

  24 months

• Annualised factor consumption

  Assessed by dispensed factor product

  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.

You may qualify if:

• Have a diagnosis of haemophilia B
• Have started Alprolix treatment prior to enrolment visit, or at enrolment prescribed treatment with Alprolix irrespective of participation in the study
• Signed and dated informed consent provided by the patient, or the patients 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 (29)

Swedish Orphan Biovitrum Research Site

Brno, Czechia

Location

Swedish Orphan Biovitrum Research Site

Prague, Czechia

Location

Swedish Orphan Biovitrum Research Site (Haemophilia Center Aghia Sophia Children's Hospital)

Athens, Greece

Location

Swedish Orphan Biovitrum Research Site (Laikο General Hospital of Athens)

Athens, Greece

Location

Swedish Orphan Biovitrum Research Site

Dublin, Ireland

Location

Swedish Orphan Biovitrum Research Site

Bari, Italy

Location

Swedish Orphan Biovitrum Research Site

Florence, Italy

Location

Swedish Orphan Biovitrum Research Site

Genova, Italy

Location

Swedish Orphan Biovitrum Research Site

Milan, Italy

Location

Swedish Orphan Biovitrum Research Site

Roma, Italy

Location

Swedish Orphan Biovitrum Research Site

Turin, Italy

Location

Swedish Orphan Biovitrum Research Site

Oslo, Norway

Location

Swedish Orphan Biovitrum Research Site (King Faisal Hospital adult)

Riyadh, Saudi Arabia

Location

Swedish Orphan Biovitrum Research Site (King Faisal Hospital pediatric)

Riyadh, Saudi Arabia

Location

Swedish Orphan Biovitrum Research Site (Hospital de Vall d'Hebrón)

Barcelona, Spain

Location

Swedish Orphan Biovitrum Research Site (Hospital Sant Joan de Deu)

Barcelona, Spain

Location

Swedish Orphan Biovitrum Research Site

Murcia, Spain

Location

Swedish Orphan Biovitrum Research Site

Vigo, Spain

Location

Swedish Orphan Biovitrum Research Site

Gothenburg, Sweden

Location

Swedish Orphan Biovitrum Research Site

Malmo, Sweden

Location

Swedish Orphan Biovitrum Research Site (Karolinska University Hospital adult)

Stockholm, Sweden

Location

Swedish Orphan Biovitrum Research Site (Karolinska University Hospital pediatric)

Stockholm, Sweden

Location

Swedish Orphan Biovitrum Research Site

Hull, United Kingdom

Location

Swedish Orphan Biovitrum Research Site

London, NW3 2QG, United Kingdom

Location

Swedish Orphan Biovitrum Research Site

London, SE1 7EH, United Kingdom

Location

Swedish Orphan Biovitrum Research Site

London, W12 0HS, United Kingdom

Location

Swedish Orphan Biovitrum Research Site

London, WC1N 3JH, United Kingdom

Location

Swedish Orphan Biovitrum Research Site

Manchester, United Kingdom

Location

Swedish Orphan Biovitrum Research Site

Oxford, United Kingdom

Location

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: April 2, 2019
• First Posted: April 3, 2019
• Study Start: September 12, 2019
• Primary Completion: March 12, 2024
• Study Completion: March 12, 2024
• Last Updated: September 29, 2025

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

ES (4); SA (2); IE (1); NO (1); IT (6); CZ (2); GR (2); GB (7); SE (4)