A 48-Month Study to Evaluate Long-Term Effectiveness of Elocta on Joint Health

  • STATUS
    Recruiting
  • End date
    Dec 12, 2025
  • participants needed
    300
  • sponsor
    Swedish Orphan Biovitrum
Updated on 12 May 2021
Investigator
Sofia Bergenstråle
Primary Contact
Kinder- und Jugendarzt-Praxis Blaubeuren (0.0 mi away) Contact
+119 other location

Summary

This is a 48-month observational, prospective, multicentre study. The overall aim of the study is to evaluate the long-term effectiveness of Elocta treatment on joint health in patients treated prophylactically with Elocta in a real-world setting.

Description

Haemophilia A is a rare genetic disorder estimated to occur in one out of 10,000 live births, characterized by a deficiency in coagulation factor VIII causing impaired haemostasis and prolonged bleeding episodes. Moderate haemophilia, defined as < 5%, and severe haemophilia, defined as < 1% of normal factor VIII activity result in frequent and spontaneous bleeds into muscles and joints, commonly the elbows, knees, and ankles. Bleeding into joints can cause acute pain and swelling and can result in reduced joint range of motion, long-term cartilage damage and debilitating haemophilic arthropathy. Early use of prophylaxis with factor VIII replacement is recommended following diagnosis of haemophilia A to maintain joint health and prevent joint destruction. However, despite the use of prophylaxis many patients still experience joint bleeds which may lead to joint deterioration over time. The risk of joint bleeds increases with the amount of time spent below certain FVIII trough levels, e.g. 1, 3 or 5 IU/dL. Thus, there is probably a relation between the intensity of the prophylactic treatment regimen and joint health. Elocta is an extended half-life rFVIII product (EHL rFVIII), with a slower clearance as compared to conventional FVIII products. Treatment with Elocta will therefore provide the treater with a greater flexibility for individualizing prophylaxis as compared to conventional FVIII. Higher trough levels can be reached with Elocta without increasing factor usage or injection frequency. The treater can instead choose to reduce the injection frequency or the factor consumption without lowering trough levels.

The purpose of this study is to evaluate the effectiveness of Elocta on joint health over a long observation period (48 months). The study will explore the influence on long term joint health of different Elocta prophylaxis regimens leading to different trough levels.

Details
Condition Hemophilia A, Hemophilia, hema
Treatment ELOCTA
Clinical Study IdentifierNCT04293523
SponsorSwedish Orphan Biovitrum
Last Modified on12 May 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Provided signed and dated informed consent by the patient, or the patient's legally authorized representative(s) for patients under the legal age, before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations
Have a diagnosis of haemophilia A
At enrolment on prophylactic treatment with Elocta, independent of participation in the study

Exclusion Criteria

Enrolment in another concurrent clinical interventional study, or intake of an Investigational Medicinal Product (IMP), within three months prior to inclusion in this study
Presence of factor VIII antibodies (inhibitors) (0.60 Bethesda Unit [BU]/mL) at the latest available inhibitor test
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