View clinical trials related to Hemophilia.
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 study investigators are interested in learning more about how drugs, that are given to children by their health care provider, act in the bodies of children and young adults in hopes to find the most safe and effective dose for children. The primary objective of this study is to evaluate the PK of understudied drugs currently being administered to children per SOC as prescribed by their treating provider.
Background. The recurrence of hemarthrosis in patients with hemophilia favors the development of an intra-articular, degenerative and progressive lesion (hemophilic arthropathy). Pain is one of the main clinical manifestations of this arthropathy. Objective. Assess the safety and efficacy of an intervention in pain through visualization of the movement using a mobile application of augmented reality and immersive 360º video, regarding the frequency of bleeding and the improvement of pain perception and quality of life, joint state , strength and functionality in patients with hemophilic arthropathy. Study design. Randomized, multicenter, simple blind clinical study. Method. 140 patients with hemophilia A and B will be recruited in this study. Patients will be recruited in 5 regions of Spain. The 8 dependent variables will be: frequency of bleeding (self-registration), pain (measured with the visual analogue scale and pressure algometer, Tampa Scale of Kinesiophobia and Pain Catastrophizing Scale), quality of life (SF-12 scale), anxiety (questionnaire Anxiety State-Trait-STAI), joint state (Hemophilia Joint Health Score scale), muscular strength (dynamometer) and functionality (6-Minutes Walking test, Quick Disabilities of the arm, shoulder and hand). Three evaluations will be carried out: pre-treatment, post-treatment and after a 6-month follow-up period. Expected results. Observe the effectiveness of movement visualization in the characteristics of pain, and its implication in functionality, joint state, muscular strength and the perception of quality of life in patients with hemophilic arthropathy.
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.
Objectives: To assess the efficacy of performing a therapeutic exercise program, compared with usual care, on pain relief, range of movement and self-perceived quality of life in patients with hemophilia Design: Quantitative, experimental, longitudinal and prospective study. Subjects: Male patients between 30 and 45 years old, with hemophilia type A or B and with knee, ankle or elbow arthropathy caused by hemophilia. Participants may be undergoing a pharmacologic treatment with intravenous VIII and/or IX factor Methods: Participants in the experimental group will an intervention based on therapeutic exercise. One therapist will instruct the patients during two sessions (each lasting approximately 1 hour). Participants will be asked to continue this program at least twice a week. Meanwhile, those in the control group will receive usual physical therapy care, based on a more passive approach, including self-assisted joint mobilization and muscle stretching. Those in the control group will be also instructed to perform the protocol at home, at least twice a week. The intervention protocol in both groups will last three months
The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median annualized spontaneous bleeding rate (AsBR) of 0.00. The majority (99%) of bleeding episodes were managed successfully with one or two infusions, and 94% of bleeds were controlled with only one infusion regardless of the cause or location. The rationale for this study is to collect the effectiveness and safety data of severe hemophilia B patients treated with Idelvion following institutional standard of care. It is the aim of this study to extend the results of the clinical trial program to a broader hemophilia B population, and to compare with current alternative factor IX treatments in Taiwan.
Hemophilia A and B are bleeding disorders caused by deficiency of factor VIII and IX, respectively. The deficiency of one of these coagulation factors is due to a mutation on the X chromosome. Accordingly replacement of the deficient factor is currently the main treatment for these disorders. The most disappointing complication of replacement therapy in hemophilia is the development of inhibitors. Unlike haemophilia , inhibitor development in patients with V Willebrand's Disease (VWD) is a rare complication of treatment. Studies on inhibitors whether on hemophilia or VWD are limited in our region. This study aims to 1. To estimate the frequency of factor inhibitors in hemophilia and VWD patients in our region. 2. To investigate modifiable risk factors associated with development of inhibitors in both diseases. 3. To correlate the level of inhibitor with the clinical presentation of the patients. 4. To assess influence of factor inhibitors on quality of life in patients who developed factor inhibitors in both diseases.
This study will be used to assess the feasibility and sensitivity of using the iSTEP, to assess exercise capacity in boys with haemophilia. The feasibility criteria to be explored includes: 1. Recruitment to target number or better 2. The test procedure is completed within an allocated time (1-2 hours) and by 90% of participants without serious adverse events 3. 90% of participants achieve a sufficient exercise response (85% maximum heart rate (HRmax)) 4. Calculation of estimates of minimum clinically important differences and variability for sample size calculations and responsiveness to severity of haemophilia and orthopaedic status The iSTEP exercise test will be compared to a more commonly used exercise test the modified shuttle walk test (10m- MSWT). Muscle strength (using myometry) will also be tested and compared to exercise performance for any relationships. Physical activity levels will also be assessed to obtain some baseline measurements of physical activity levels in this patient population, which are currently not very well known. The overarching aim of this current study is also to establish a robust and sensitive exercise test and to gain an understanding of the effects of physical activity levels and muscle strength on exercise capacity in this population.
The purpose of this study is to investigate the safety of a test drug to treat hemophilia in adult men.
Primary Objective: - To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B Secondary Objectives: - To characterize the safety and tolerability - To determine fitusiran plasma concentrations at selected time points