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

NCT ID: NCT02976753

Last Updated: 2024-10-02

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

361 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-12-31

Study Completion Date

2021-11-03

Brief Summary

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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.

Detailed Description

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Conditions

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Hemophilia A

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Elocta

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

efmoroctocog alfa

Intervention Type DRUG

extended half-life factor VIII product

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

Factor VIII

Intervention Type DRUG

conventional factor VIII product

Interventions

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efmoroctocog alfa

extended half-life factor VIII product

Intervention Type DRUG

Factor VIII

conventional factor VIII product

Intervention Type DRUG

Other Intervention Names

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Elocta

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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
Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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ICON plc

INDUSTRY

Sponsor Role collaborator

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Physician, MD

Role: STUDY_DIRECTOR

Sweden Orphan Biovitrum

Locations

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Swedish Orphan Biovitrum Research site

Innsbruck, , Austria

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Linz, , Austria

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Sankt Pölten, , Austria

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Vienna, , Austria

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Brussels, , Belgium

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Helsinki, , Finland

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Caen, , France

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Montpellier, , France

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Nancy, , France

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Rennes, , France

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Catanzaro, , Italy

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Florence, , Italy

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Milan, , Italy

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Naples, , Italy

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Padua, , Italy

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Palermo, , Italy

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Parma, , Italy

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Rome, , Italy

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Turin, , Italy

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Oslo, , Norway

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Alicante, , Spain

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Barcelona, , Spain

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Madrid, , Spain

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Málaga, , Spain

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Seville, , Spain

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Valencia, , Spain

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Gothenburg, , Sweden

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Malmo, , Sweden

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Stockholm, , Sweden

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Birmingham, , United Kingdom

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Canterbury, , United Kingdom

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Liverpool, , United Kingdom

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London, , United Kingdom

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Manchester, , United Kingdom

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Sheffield, , United Kingdom

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Swedish Orphan Biovitrum Research site

Southampton, , United Kingdom

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Countries

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Austria Belgium Finland France Italy Norway Spain Sweden United Kingdom

References

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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.

Reference Type DERIVED
PMID: 40616274 (View on PubMed)

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.

Reference Type DERIVED
PMID: 31152037 (View on PubMed)

Other Identifiers

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Sobi.Elocta-002

Identifier Type: -

Identifier Source: org_study_id

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