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

NCT ID: NCT04293523

Last Updated: 2025-01-22

Basic Information

• Recruitment Status: ACTIVE_NOT_RECRUITING
• Total Enrollment: 427 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2020-03-30
• Study Completion Date: 2026-04-30

Brief 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.

Detailed 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.

Patients may limit their physical activities due to fear of bleeding if they are unaware of their current FVIII level. Patient apps and wearables are now available which allow patients to view their predicted FVIII levels, and capture health-related data (such as bleedings, pain, well-being, physical activity levels etc.). This data can be shared with the treating physician supporting the planning to individualize the patient's factor treatment based on current lifestyle, health status and physical activity levels. Florio, a certified medical device used as part of routine clinical practice, is such an app, and the data output and patient feedback on their activity levels and sense of protection while using Florio will be analysed as exploratory objectives in this study.

The main purpose of this study is to evaluate the effectiveness of Elocta on joint health over a long observation period (48 months). The study will also explore the influence on long term joint health of different Elocta prophylaxis regimens leading to different trough levels and if the extent of patients' physical activity levels can be associated with predicted FVIII levels.

Conditions

Hemophilia A

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Hemophilia A patients

All patients diagnosed with haemophilia A regardless of severity, on factor treatment with Elocta according to usual clinical practice.

ELOCTA

Intervention Type: DRUG

Extended half-life factor VIII product

Interventions

ELOCTA

Extended half-life factor VIII product

Intervention Type: DRUG

Other Intervention Names

ELOCTATE; efmoroctocog alfa; rFVIIIFc

Eligibility Criteria

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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Physician

Role: STUDY_DIRECTOR

Swedish Orphan Biovitrum AB

Locations

University Hospital Brno

Brno, , Czechia

University Hospital Ostrava

Ostrava, , Czechia

Dept. of Pediatric Haematology and Oncology, University Hospital Motol

Prague, , Czechia

Lastehaigla, Tallinn (Tallinn Children´s Hospital)

Tallinn, , Estonia

The North Estonia Medical Centre Hematoloogiakeskus, Regionalhaigla

Tallinn, , Estonia

Helsinki University Central Hospital, New Children Hospital

Helsinki, , Finland

Turku University Central Hospital, Paediatric and adolescent haematology and oncology clinic

Turku, , Finland

Charité-Universitätsmedizin Berlin Campus Virchow Klinikum

Berlin, , Germany

Universitätsklinikum Bonn AöR, Institut für Experimentelle Hämatologie und Transfusionsmedizin

Bonn, , Germany

Hämostaseologie/Hämophiliezentrum, Medizinische Klinik 2 Institut für Transfusionsmedizin, Universitätsklinikum

Frankfurt, , Germany

Universitätsklinikum Frankfurt - Klinik für Kinder- und Jugendmedizin

Frankfurt, , Germany

Universitätsklinikum Hamburg-Eppendorf (UKE)

Hamburg, , Germany

Medizinische Hochschule Hannover

Hanover, , Germany

Werlhof-Institut für Hämostaseologie GmbH

Hanover, , Germany

SRH-Klinikum Heidelberg

Heidelberg, , Germany

HZRM Hämöphilie Zentrum Rhein Main

Mörfelden-Walldorf, , Germany

Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital am Universitätsklinikum München

München, , Germany

Ippokrateio Hospital Thessaloniki (adult department)

Thessaloniki, , Greece

Ippokrateio Hospital Thessaloniki (pediatric department)

Thessaloniki, , Greece

University of Bari Aldo Moro (Centro Emofilia Policlinico - Pediatria U.O.)

Bari, , Italy

AUSL Romagna Centro Emofilia U.O.C., Medicina Trasfusionale Dipartimento Patologia, Clinica Ospedale M. Bufalini

Cesena, , Italy

Giannina Gaslini Institute

Genova, , Italy

Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, , Italy

University Hospital of Parma, AOUP, Haemophilia Center

Parma, , Italy

Uo Malattie Emorragiche e Trombotiche Fondazione Policlinico Universitario Agostino Gemelli, IRCCS, Universitá Catolica del Sacro Coure

Rome, , Italy

Azienda Ospedaliera Città della Salute e della Scienza di Torino Regina Margherita

Torino, , Italy

University Medical Center Groningen/UMCG

Groningen, , Netherlands

The Royal Hospital

Muscat, , Oman

Dr Suliman Al Habib Hospital Riyadh

Riyadh, , Saudi Arabia

King Faisal Specialised Hospital, KFSH Riyadh, Children

Riyadh, , Saudi Arabia

King Faisal Specialist Hospital KFSH, Adults

Riyadh, , Saudi Arabia

Riyadh Military Hospital (P.S.M.C)

Riyadh, , Saudi Arabia

University Medical Centre Ljubljana Division of Paediatrics

Ljubljana, , Slovenia

Hospital de la Santa Creu i Sant Pau

Barcelona, , Spain

Hospital Vall d'Hebron

Barcelona, , Spain

Sant Johan De Deu

Barcelona, , Spain

Hospital Universitario Cruces

Cruces, , Spain

Hospital Universitario Donostia

Donostia / San Sebastian, , Spain

Hospital Universitario Carlos Haya

Málaga, , Spain

Hospital Virgen de la Arrixaca

Murcia, , Spain

Complejo Hospitalario de Navarra

Navarro, , Spain

Hospital Universitario Central de Asturias (HUCA)

Oviedo, , Spain

Hospital Universitario Son Espases

Palma de Mallorca, , Spain

Hospital Universitario Virgen del Rocío

Seville, , Spain

Hospital Alvaro Cunqueiro

Vigo, , Spain

Hematologimottagning Sahlgrenska

Gothenburg, , Sweden

Department of Haematology, Oncology and Radiation Physics, Skåne University Hospital Malmö

Malmo, , Sweden

Universitätsklinik für Hämatologie und Hämatologisches Zentrallabor Inselspital

Bern, , Switzerland

Service et Laboratoire central d'hématologie, Adults

Lausanne, , Switzerland

Zentrum für Labormedizin

Sankt Gallen, , Switzerland

Universitätsspital Zürich Klinik für Medizinische Onkologie und Hämatologie

Zurich, , Switzerland

East Kent Hospitals University NHS Foundation Trust, Kent Haemophilia and Thrombosis Centre, Kent and Canterbury Hospital

Canterbury, , United Kingdom

Great Ormond Street Hospital, Royal London Hospital for Integrated Medicine

London, , United Kingdom

Countries

Czechia; Estonia; Finland; Germany; Greece; Italy; Netherlands; Oman; Saudi Arabia; Slovenia; Spain; Sweden; Switzerland; United Kingdom

Other Identifiers

Sobi.Elocta-005

Identifier Type: -

Identifier Source: org_study_id