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CompletedNCT02976753Updated Oct 2, 2024

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

An observational study in Hemophilia A, sponsored by Swedish Orphan Biovitrum. Completed at 36 sites in 9 countries. Open to male participants. Per ClinicalTrials.gov, last updated 2024-10-02.

Sponsored by Swedish Orphan Biovitrum · Observational

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
361
Sex
Male
01

Study 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.

02

Conditions studied

  • Hemophilia A

Keywords

  • Blood Coagulation Disorders
  • Hematologic Diseases
  • Coagulation Protein Disorders
  • Hemorrhagic Disorders
  • Genetic Diseases, Inborn
03

In context

Hemophilia A

885 studies on the registry are indexed under Hemophilia A; 139 are open to participants now.

This study's enrollment of 361 is above the median of 80 across 322 observational studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Swedish Orphan Biovitrum is the lead sponsor of 80 studies on the registry; 4 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 15 (88%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
Male
Accepts healthy volunteers
No
Sampling method
Probability sample

Study population

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

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

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
05

Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
361 participants (actual)
Patient registry
No

Groups and cohorts

  • 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

06

What researchers measure

Primary outcomes

  1. Annualised bleeding rate (ABR)

    Time frame: 24 months

  2. Annualised injection frequency

    Time frame: 24 months

  3. Annualised factor consumption (International Unit [IU])

    Time frame: 24 months

07

Study locations

36 sites
  • Swedish Orphan Biovitrum Research site
    Innsbruck, Austria
  • Swedish Orphan Biovitrum Research site
    Linz, Austria
  • Swedish Orphan Biovitrum Research Site
    Sankt Pölten, Austria
  • Swedish Orphan Biovitrum Research site
    Vienna, Austria
  • Swedish Orphan Biovitrum Research site
    Brussels, Belgium
  • Swedish Orphan Biovitrum Research site
    Helsinki, Finland
  • Swedish Orphan Biovitrum Research site
    Caen, France
  • Swedish Orphan Biovitrum Research site
    Montpellier, France
  • Swedish Orphan Biovitrum Research site
    Nancy, France
  • Swedish Orphan Biovitrum Research site
    Rennes, France
  • Swedish Orphan Biovitrum Reserach site
    Catanzaro, Italy
  • Swedish Orphan Biovitrum Reserach site
    Florence, Italy
  • Swedish Orphan Biovitrum Research site
    Milan, Italy
  • Swedish Orphan Biovitrum Reserach site
    Naples, Italy
  • Swedish Orphan Biovitrum Research Site
    Padova, Italy
  • Swedish Orphan Biovitrum Reserach site
    Palermo, Italy
  • Swedish Orphan Biovitrum Reserach site
    Parma, Italy
  • Swedish Orphan Biovitrum Reserach site
    Rome, Italy
  • Swedish Orphan Biovitrum Research site
    Turin, Italy
  • Swedish Orphan Biovitrum Research site
    Oslo, Norway
  • Swedish Orphan Biovitrum research site
    Alicante, Spain
  • Swedish Orphan Biovitrum Reserach site
    Barcelona, Spain
  • Swedish Orphan Biovitrum Reserach site
    Madrid, Spain
  • Swedish Orphan Biovitrum Research site
    Málaga, Spain
  • Swedish Orphan Biovitrum Reserach site
    Seville, Spain
  • Swedish Orphan Biovitrum Research site
    Valencia, Spain
  • Swedish Orphan Biovitrum Research site
    Gothenburg, Sweden
  • Swedish Orphan Biovitrum Research site
    Malmö, Sweden
  • Swedish Orphan Biovitrum Research site
    Stockholm, Sweden
  • Swedish Orphan Biovitrum Research site
    Birmingham, United Kingdom
  • Swedish Orphan Biovitrum Research site
    Canterbury, United Kingdom
  • Swedish Orphan Biovitrum Research site
    Liverpool, United Kingdom
  • Swedish Orphan Biovitrum Reserach site
    London, United Kingdom
  • Swedish Orphan Biovitrum Research site
    Manchester, United Kingdom
  • Swedish Orphan Biovitrum Research site
    Sheffield, United Kingdom
  • Swedish Orphan Biovitrum Research site
    Southampton, United Kingdom
08

References and documents

Publications

  • 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. PubMed 40616274 ↗
  • 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. PubMed 31152037 ↗

Individual participant data

Plan to share: Undecided

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 2, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT02976753
Collaborators
ICON plc
Responsible party
Sponsor
First posted
Nov 29, 2016
Start date
Dec 2016
Primary completion
Nov 3, 2021
Completion
Nov 3, 2021
Last update
Oct 2, 2024

Study contacts

Study Physician, MD
study director · Sweden Orphan Biovitrum

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.

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