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Clinical Trial Details — Status: Active, not recruiting

Administrative data

NCT number NCT04293523
Other study ID # Sobi.Elocta-005
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date March 30, 2020
Est. completion date April 2026

Study information

Verified date April 2024
Source Swedish Orphan Biovitrum
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial 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. 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.


Recruitment information / eligibility

Status Active, not recruiting
Enrollment 427
Est. completion date April 2026
Est. primary completion date April 2026
Accepts healthy volunteers No
Gender All
Age group N/A and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
ELOCTA
Extended half-life factor VIII product

Locations

Country Name City State
Czechia University Hospital Brno Brno
Czechia University Hospital Ostrava Ostrava
Czechia Dept. of Pediatric Haematology and Oncology, University Hospital Motol Praha
Estonia Lastehaigla, Tallinn (Tallinn Children´s Hospital) Tallinn
Estonia The North Estonia Medical Centre Hematoloogiakeskus, Regionalhaigla Tallinn
Finland Helsinki University Central Hospital, New Children Hospital Helsinki
Finland Turku University Central Hospital, Paediatric and adolescent haematology and oncology clinic Turku
Germany 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
Greece Ippokrateio Hospital Thessaloniki (adult department) Thessaloníki
Greece Ippokrateio Hospital Thessaloniki (pediatric department) Thessaloníki
Italy 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
Netherlands University Medical Center Groningen/UMCG Groningen
Saudi Arabia 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
Slovenia University Medical Centre Ljubljana Division of Paediatrics Ljubljana
Spain 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
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 Sevilla
Spain Hospital Alvaro Cunqueiro Vigo
Sweden Hematologimottagning Sahlgrenska Gothenburg
Sweden Department of Haematology, Oncology and Radiation Physics, Skåne University Hospital Malmö Malmö
Switzerland Universitätsklinik für Hämatologie und Hämatologisches Zentrallabor Inselspital Bern
Switzerland Service et Laboratoire central d'hématologie, Adults Lausanne
Switzerland Zentrum für Labormedizin Saint Gallen
Switzerland Universitätsspital Zürich Klinik für Medizinische Onkologie und Hämatologie Zürich
United Kingdom 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

Sponsors (1)

Lead Sponsor Collaborator
Swedish Orphan Biovitrum

Countries where clinical trial is conducted

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

Outcome

Type Measure Description Time frame Safety issue
Other Effectiveness of Elocta: Annualised bleeding rate (ABR) for treated bleeds Number of bleeding events per year, for treated bleeds 48 months
Other Effectiveness of Elocta: Annualised bleeding rate (ABR) for all bleeds Number of bleeding events per year, for all bleeds 48 months
Other Effectiveness of Elocta: Occurrence of zero (0) joint bleeds Percentage (%) of study population with zero (0) joint bleeds 48 months
Other Effectiveness of Elocta: Work productivity and impairment Assessed by WPAI-SHP questionnaire 48 months
Other Effectiveness of Elocta: Quality of Life Assessed by Haemo-QoL/Haem-A-QoL questionnaire 48 months
Other Effectiveness of Elocta: Physical activity level Assessed by the Saltin-Grimby scale 48 months
Other Effectiveness of Elocta: Quality of Life Assessed by EQ-5D-5L/EQ-5D-Y questionnaire 48 months
Other Effectiveness of Elocta: Severity of joint health Assessed by Patient Global Impression of Severity of Joint Health questionnaire 48 months
Other Effectiveness of Elocta: FVIII plasma levels Percentage (%) of FVIII plasma level 48 months
Other Effectiveness of Elocta: Usage of pain and anti-inflammatory medication Assessed by medication dose 48 months
Other Effectiveness of Elocta: Usage of pain and anti-inflammatory medication Assessed by total number of exposure days per medication 48 months
Other Usage of Elocta: Annualised injection frequency per subject Assessed by prescription 48 months
Other Usage of Elocta: Annualised factor consumption per subject Assessed by prescription [IU/kg] 48 months
Other Usage of Elocta: Adherence Investigator assessed (Percentage (%)) 48 months
Other Exploratory Objective Influence of different Elocta prophylaxis regimens on long term joint health 48 months
Other Exploratory Objective: Impact of florio HAEMO on patients' sense of protection and their activity level Results from questionnaire on impact of florio HAEMO tools on patients' sense of protection and their activity level 48 months
Other Exploratory Objective: Explore adherence to prescribed treatment regimen florio HAEMO captured treatment adherence scores 48 months
Other Exploratory Objective: Explore relationship between predicted FVIII levels and physical activity florio HAEMO captured predicted FVIII levels, florio HAEMO captured physical activity (type, duration, heart rate and steps) 48 months
Other Exploratory Objective: Explore timing of bleeds in relation to predicted FVIII levels and physical activity florio HAEMO captured bleed data (timing, location, cause, treated/untreated), florio HAEMO captured injection data (time, dose), florio HAEMO captured predicted FVIII levels 48 months
Other Exploratory Objective: Explore the use of Florio to characterise pain and well-being florio HAEMO captured pain data (time, location, cause, intensity), florio HAEMO captured well-being data 48 months
Other Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health Target joint development, resolution and recurrence 48 months
Other Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health Annualised joint bleeding rate (AJBR) for treated bleeds 48 months
Other Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health Ultrasound (HEAD-US) 48 months
Other Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health Clinical joint scoring (HJHS) 48 months
Other Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health Annualised bleeding rate (ABR) (based on bleeding episodes assessed according to local practice) for all bleeds 48 months
Other Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health Occurrence of zero (0) joint bleeds 48 months
Other Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health Quality of Life assessed by Haemo-QoL/Haem-A-QoL 48 months
Other Exploratory Objective: Influence of different Elocta prophylaxis regimens on long term joint health Physical activity level assessed by the Saltin-Grimby scale 48 months
Primary Joint health: Target joint development Number of target joints 48 months
Primary Joint health: Target joint resolution Number of resolved target joints 48 months
Primary Joint health: Target joint recurrence Number of recurring target joints 48 months
Primary Joint health: Annualised joint bleeding rate (AJBR) for treated bleeds Number of joint bleeding events per year, for treated bleeds 48 months
Secondary Disease Activity (hypertrophic synovium) and Disease Damage (Cartilage or Bone) scores for elbows, knees and ankles The Haemophilia Early Arthropathy Detection with UltraSound (HEAD-US) protocol will be used. The total score represents the sum of item scores for abnormalities detected. Its values range from 0 (minimum) to 8 (maximum). A higher score indicates a worse outcome. 48 months
Secondary Global Gait Score, and/or joint score items for elbows, knees and ankles. The Haemophilia Joint Health Score (HJHS) system will be used. Global Gait Score, and/or Joint score items (swelling, duration of swelling, muscle atrophy, axial alignment, crepitus on motion, flexion loss, instability, extension loss, joint pain, strength, gait) for elbows, knees and ankles.
The minimum score per joint is 0, the maximum score is 20. The overall total joint score (range 0-120) is the sum of the 6 six index joint (elbows, knees and ankles) scores. A higher score indicates a worse outcome.
48 months
Secondary Joint and physical evaluation for elbows, knees and ankles. The WFH Physical Examination Score (AKA Gilbert Score) will be used. Joint evaluation (pain, bleeding, physical examination and radiologic evaluation) and physical evaluation (swelling, muscle atrophy, axial deformity, crepitus on motion, range of motion, flexion contracture, and instability). A higher score indicates a worse outcome. 48 months
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