View clinical trials related to Hemophilia A.
Filter by:The improvements observed in the care of patients with hemophilia or Willebrand disease have led to an increase in their life expectancy, which today approaches that of the general population. This increase in life expectancy leads in these patients to the development of comorbidities related to aging (cardiovascular and neurological diseases, cancers and kidney diseases) (e.g "Franchini & Mannuccio", BJH, 2009). The care of these comorbidities represents a new challenge for the medical teams. Toward multiple comorbidities, polypharmacy is often associated. Many studies about medication exposure and management in older patients were published but no study was conducted to explore the medication management of older patients with hemophilia or Willebrand disease.
The investigators propose, as part of the study, to carry out for each patient: - An analysis of monocytic populations by flow cytometry (CD14, CD16, CD45, CD68, CD115, CCR2, CX3CR1, CD163 and CD206). - A population assessment of Myeloid-Derived Suppressor Cells (MDSC). - Assays of cytokines and chemokines involved in inflammation by multiplex analyzes: Il-1 (α and β), Il-4, Il-6, Il-10, Il-13, TNF- α, TGF- β, CRP , leptin, IFN- β. - Specialized dosages of proteins involved in bone metabolism. RANKL, osteoprotegerin, M-CSF, TRAPCP5.
this study evaluates the effects of therapeutic exercises on kinesiophobia and health-related quality of life in adult haemophilia patients. half of participants will receive therapeutic exercises and verbal information about the positive effects of therapeutic exercises on physical pathologies due to hemophilic arthropathy while the other half will receive only verbal information.
Hemophilia A is a severe, life-long, genetic bleeding disorder characterized by a deficiency of factor VIII (FVIII), a crucial cofactor of the coagulation system. The mainstay of hemophilia treatment is factor replacement therapy with FVIII clotting factor concentrates (CFC) and these can be given episodically in response to bleeding or prophylactically to prevent bleeding. The main adverse effect of FVIII CFC is the development of neutralizing anti-drug antibodies termed inhibitors, and these render replacement therapy less effective if they are low titer inhibitors or completely ineffective if they are of the high titer variety. These so-called 'inhibitor patients' cannot rely on FVIII CFC for their treatment and are treated with other CFC called bypassing agents such as activated prothrombin complex concentrate (aPCC/Feiba). While these agents can be effective in some patients for prophylaxis, they are not as effective for bleed prevention as FVIII CFC for patients without inhibitors.Recently, emicizumab (Hemlibra, Roche), was developed and licensed for the prevention of bleeding in patients with hemophilia A with and without inhibitors. However, patients in the clinical trials for emicizumab have developed thrombotic adverse events and only patients who received doses of Feiba of >100 IU/kg/24 hours for more than 24 hours developed thrombosis. As a result of the above data, recommendations have been to either avoid altogether in patients on emicizumab, or to be very cautious about using it to treat breakthrough bleeding. With this in mind, we propose to study the in vivo combination of Feiba in patients with inhibitors on emicizumab.
This study is investigating how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medication that will be used for prevention of bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected with a thin needle in the skin of the stomach, using a pen-injector. The study will last for up to 44 months. It consists of a main phase (part 1 and part 2) and an extension phase. In part 1, participants will be injected only once with either Mim8 or a "dummy" medicine (placebo) - which one will be decided by chance. In part 2 and the extension phase participants will get an Mim8 injection weekly or monthly.
This study is an international, multicenter, open-label, single arm, prospective clinical trial and will evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).
The study aims to describe physical activity (PA) levels in young people with haemophilia A in Norway compared with non-haemophilia controls, and to identify factors influencing PA. This will be conducted through an observational study measuring PA over 12 weeks. Forty young people with moderate and severe haemophilia A will be enrolled. PA data will be compared to demographically and seasonally matched non-haemophilia controls. PA will be measured using the activity tracker Fitbit Charge 3. A subgroup of participants will also wear the hip-worn accelerometer ActiGraph GTX+BT for seven consecutive days in order to validate the two devices against each other.
This multicenter, non-interventional, prospective study will collect information about activity status, bleeds, health-related quality of life (HRQoL), health status, and safety in participants with moderate or severe haemophilia A without factor VIII (FVIII) inhibitors, who are being treated in accordance with normal clinical practice.
Primary Objective: - To evaluate the efficacy of BIVV001 as a prophylaxis treatment in prophylaxis treatment arm. Secondary Objectives: - To evaluate the efficacy of BIVV001 as a prophylaxis treatment. - To evaluate the efficacy of BIVV001 in the treatment of bleeding episodes. - To evaluate BIVV001 consumption for the prevention and treatment of bleeding episodes. - To evaluate the effect of BIVV001 prophylaxis on joint health outcomes. - To evaluate the effect of BIVV001 prophylaxis on Quality of Life outcomes. - To evaluate the efficacy of BIVV001 for perioperative management. - To evaluate the safety and tolerability of BIVV001 treatment. - To assess the pharmacokinetics (PK) of BIVV001 based on the 1-stage activated partial thromboplastin time (aPTT) and 2-stage chromogenic coagulation factor VIII (FVIII) activity assays.
The main aim of this study is to check for long-term side effects from ADYNOVI/ADYNOVATE prophylaxis in participants with haemophilia A when used under standard clinical practice in the real-world clinical setting.