Joint Health Study
Research type
Research Study
Full title
Non-Interventional Study of the Change in Joint Health in Adult Patients with Haemophilia A after Switching to Prophylaxis with Turoctocog Alfa Pegol (N8-GP)
IRAS ID
316218
Contact name
Deepika Singh Panwar
Contact email
Sponsor organisation
Novo Nordisk Service Centre India Private Ltd.
Clinicaltrials.gov Identifier
52917, CPMS ID
Duration of Study in the UK
2 years, 6 months, 2 days
Research summary
Study aimed to investigate the effect of Esperoct® prophylaxis on long term patient joint health in persons with haemophilia A in a real-world setting.
Haemophilia A is a congenital disorder caused by mutations in the coagulation factor VIII gene. Deficiency of coagulation factor VIII impairs clotting. That leads to bleeding episodes - spontaneous or after mild trauma in muscles, soft tissues, and joints. Recurrent bleeding in the same location, particularly in the major joints, may lead to a condition known as target joint. Left unchecked, this may lead to muscular thinning and deformities.
Joint health is considered by patients, doctors, and public health bodies to be one of the most relevant factors affecting quality of life. Joint health is a major factor in the burden of haemophilia treatment, influencing treatment costs and patient productivity. Joints impairment can be quantified using the Haemophilia Joint Health Score version 2.1, a validated clinical assessment tool.
Efficacy and safety profile of Esperoct® for the treatment of patients with haemophilia A is well examined in pivotal-phase 3 trials. Information is limited regarding the impact of Esperoct® prophylaxis on long term joint health. This study assesses the change in joint health after switching to Esperoct® prophylaxis in patients with haemophilia A.Study will be conducted at NHS Sites, specialized on haemophilia management, and will enroll patients with severe or moderate haemophilia A, 18 years of age or older, switched to prophylaxis treatment with Esperoct® from previous therapy. Patient`s participation in the study will last up to 24 months and will include collection of clinical data per routine clinical practice at baseline, intermediate and end of study visits. Also, biannual remote direct data capture by participants via completing patient reported outcomes questionnaires is planned.
REC name
North West - Haydock Research Ethics Committee
REC reference
22/NW/0288
Date of REC Opinion
22 Sep 2022
REC opinion
Further Information Favourable Opinion