Prospective, Non-interventional Study to Evaluate the Effectiveness of Elocta Compared to Conventional Factor Products

NCT02976753 | Completed

• 1 other identifier: Sobi.Elocta-002
• Study Type: observational
• Target: 361
• Locations: 9 countries
• Sites: 36

Brief Summary

The purpose of the study is to evaluate the effectiveness of Elocta compared to conventional factor products in the prophylactic treatment of patients with haemophilia A over a 24-month prospective period. Data will also be collected for a 12 month retrospective period.

Conditions

Hemophilia A

Keywords

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

Outcome Measures

Primary Outcomes (3)

• Annualised bleeding rate (ABR)

  24 months

• Annualised injection frequency

  24 months

• Annualised factor consumption (International Unit [IU])

  24 months

Study Arms (2)

Elocta

Elocta will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice

Drug: efmoroctocog alfa

Conventional factor VIII product

Conventional factor VIII products will be prescribed and administered for prophylactic treatment of patients with haemophilia A according to usual clinical practice

Drug: Factor VIII

Interventions

efmoroctocog alfa DRUG

extended half-life factor VIII product

Also known as: Elocta

Elocta

Factor VIII DRUG

conventional factor VIII product

Conventional factor VIII product

Eligibility Criteria

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

Study Population

Male patients with a diagnosis of haemophilia A prescribed prophylactic treatment with Elocta or conventional factor product

You may qualify if:

• Male patients with a diagnosis of haemophilia A
• Received prophylactic treatment with a factor product for management of haemophilia A in the 12 months prior to enrolment
• At enrolment prescribed prophylactic treatment with Elocta or conventional factor product regardless of participation in the study
• Having at least 12 months documented pre-study treatment data regarding prophylactic treatment prescriptions and bleeding episodes prior to the baseline visit
• 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:

• Enrolment in a concurrent clinical interventional study involving intake of an investigational medicinal product (IMP), within 1 year prior to enrolment
• Previously treated with commercially available extended half-life products other than Elocta
• Presence of factor VIII inhibitors (≥0.60 Bethesda Unit \[BU\]/mL ) at the latest available inhibitor test using the Nijmegen modified Bethesda assay

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead
• ICON plc: collaborator

Study Sites (36)

Swedish Orphan Biovitrum Research site

Innsbruck, Austria

Location

Swedish Orphan Biovitrum Research site

Linz, Austria

Location

Swedish Orphan Biovitrum Research Site

Sankt Pölten, Austria

Location

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Vienna, Austria

Location

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Brussels, Belgium

Location

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Helsinki, Finland

Location

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Caen, France

Location

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Montpellier, France

Location

Swedish Orphan Biovitrum Research site

Nancy, France

Location

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Rennes, France

Location

Swedish Orphan Biovitrum Reserach site

Catanzaro, Italy

Location

Swedish Orphan Biovitrum Reserach site

Florence, Italy

Location

Swedish Orphan Biovitrum Research site

Milan, Italy

Location

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Naples, Italy

Location

Swedish Orphan Biovitrum Research Site

Padua, Italy

Location

Swedish Orphan Biovitrum Reserach site

Palermo, Italy

Location

Swedish Orphan Biovitrum Reserach site

Parma, Italy

Location

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Rome, Italy

Location

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Turin, Italy

Location

Swedish Orphan Biovitrum Research site

Oslo, Norway

Location

Swedish Orphan Biovitrum research site

Alicante, Spain

Location

Swedish Orphan Biovitrum Reserach site

Barcelona, Spain

Location

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Madrid, Spain

Location

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Málaga, Spain

Location

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Seville, Spain

Location

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Valencia, Spain

Location

Swedish Orphan Biovitrum Research site

Gothenburg, Sweden

Location

Swedish Orphan Biovitrum Research site

Malmo, Sweden

Location

Swedish Orphan Biovitrum Research site

Stockholm, Sweden

Location

Swedish Orphan Biovitrum Research site

Birmingham, United Kingdom

Location

Swedish Orphan Biovitrum Research site

Canterbury, United Kingdom

Location

Swedish Orphan Biovitrum Research site

Liverpool, United Kingdom

Location

Swedish Orphan Biovitrum Reserach site

London, United Kingdom

Location

Swedish Orphan Biovitrum Research site

Manchester, United Kingdom

Location

Swedish Orphan Biovitrum Research site

Sheffield, United Kingdom

Location

Swedish Orphan Biovitrum Research site

Southampton, United Kingdom

Location

Related Publications (2)

• Oldenburg J, Hay C, Peyvandi F, Lehtinen AE, Pabinger I, Nuesch E, Malmstrom H, Bednar E, Lethagen S. A-SURE: intra-patient comparison of prophylactic effectiveness of a recombinant factor VIII Fc fusion protein versus standard half-life factor VIII in hemophilia A. Hematology. 2025 Dec;30(1):2513186. doi: 10.1080/16078454.2025.2513186. Epub 2025 Jul 4.

  PMID: 40616274 DERIVED

• Oldenburg J, Hay CRM, Jimenez-Yuste V, Peyvandi F, Schved JF, Szamosi J, Winding B, Lethagen S. Design of a prospective observational study on the effectiveness and real-world usage of recombinant factor VIII Fc (rFVIIIFc) compared with conventional products in haemophilia A: the A-SURE study. BMJ Open. 2019 May 30;9(5):e028012. doi: 10.1136/bmjopen-2018-028012.

  PMID: 31152037 DERIVED

MeSH Terms

Conditions

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

Interventions

factor VIII-Fc fusion protein; Factor VIII

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Protein Precursors; Biological Factors

Study Officials

• Study Physician, MD

  Sweden 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: November 24, 2016
• First Posted: November 29, 2016
• Study Start: December 1, 2016
• Primary Completion: November 3, 2021
• Study Completion: November 3, 2021
• Last Updated: October 2, 2024

Record last verified: 2024-10

Locations

IT (9); BE (1); NO (1); AU (4); FI (1); FR (4); ES (6); SE (3); GB (7)