View clinical trials related to Hemophilia.
Filter by:This study aims to develop a systematic genetic screening strategy for (potential) female carriers of haemophilia by identifying as many female carriers as possible within the families of haemophilia patients regularly followed at Cliniques universitaires Saint-Luc (CUSL) and to search for differences between female carriers of haemophilia A (HA) and B (HB).
Hemophilia (A and B) are X-linked hereditary bleeding disorders whose severity depends on the level of coagulation factor (FVIII or FIX respectively). Bleeding is mainly from joints (hemarthrosis) and muscles (hematoma). Nowadays, treatment is based on preventive or curative intravenous infusion of coagulation factor concentrates. Despite these treatments, there is joint deterioration that can be responsible for hemophilic arthropathy and chronic pain. This pain may be related to excess nociception during acute bleeding, but it may also be neuropathic. There are only a few studies that have looked at pain in hemophilia, but it is accepted that the vast majority of patients (especially those with severe forms) suffer from chronic pain. Because patients have become accustomed to this pain and physicians are still not very aware of how to assess it, this pain is not treated effectively. In order to better manage pain in hemophilia, it is therefore necessary to characterize their pain and to know the nociceptive or neuropathic component. The aim of our study is therefore to study the prevalence, the nociceptive or neuropathic profile, the chronic aspect of pain and the main locations of pain in hemophilia. In addition to raising the awareness of physicians in the centers about pain management using specific questionnaires, this survey will help to better define chronic pain in hemophilia of all severities.
Hemophilia A and B are inherited disorders characterized by deficient or missing coagulation factors VIII or IX, respectively, of which the main long-term clinical manifestation is joint damage. Patients with haemophilia (PwH) are susceptible to clinical joint bleeding that may cause irreversible joint damage. Some degree of damage may already occur after the first haemarthrosis or even in children who never experienced clinically evident joint bleeds. Joints are mechanical systems with a structure strictly related to functioning. Therefore, any alteration in structure may have an impact on function (starting from the primary level of posture and anti-gravity muscles), which might in turn stress the joints and increase the risk of bleeding.The primary aim of this study is to investigate the changes in posture and the mechanical properties of anti-gravity muscles of adolescent PwH. The secondary aim is to determine the effect of joint dysfunction on posture in adolescent PwH.
Introduction: Hemophilic ankle arthropathy is manifested by degenerative functional alterations (deficit of muscle strength, mobility and proprioception) (intra-articular alterations) and chronic pain. Myofascial release techniques are used to treat soft tissue adhesions, relieve pain and reduce tissue sensitivity. Design. A randomized clinical trial. Aimed: To evaluate the safety and effectiveness of a protocol by self-myofascial release with Foam Roller applied in patients with hemophilic ankle arthropathy. Patients: 70 patients with ankle arthropathy will be recruited for inclusion in the study. Patients will be recruited in 5 centers, from different regions of Spain. Intervention: Each session will last approximately 15 minutes, with five physiotherapy sessions per week for a period of 3 months. Patients will be evaluated at baseline, after the intervention, and after a follow-up period of 3 months. The treatment program includes 11 exercises that must be administered bilaterally. A mobile application will be developed where each patient will be able to observe the exercises to be carried out. Measuring instruments and study variables: digital goniometer (ankle range of motion); visual analog scale and pressure algometer (joint pain); Haemophilia Joint Health Score (joint status); dynamometer assess (muscle strength); 6-Minute Walking test (functionality of lower limbs); Mobile device (Activity record); Finger-floor test (muscle flexibility). At the same time, the study will allow to determine joint bleeding caused by applied physiotherapy treatment. Expected results: To demonstrate the safety of this Physiotherapy technique in patients with hemophilia. Likewise, an improvement in ankle pain, functionality and joint motion is expected.
Background. The main physical sequela of patients with hemophilia is the development of a progressive, degenerative intra-articular lesion, known as hemophilic arthropathy). This sequela is manifested by chronic pain, limited range of motion, axial abnormalities, and periarticular muscle atrophy. Objective. To assess the safety and effectiveness of an intervention through blood flow restriction, regarding the frequency of bleeding and the improvement in the perception of muscle activation and strength, functionality, joint pain, joint status and the perception of quality of life in patients with hemophilic arthropathy. knee and ankle. Study design. Randomized, multicenter, single-blind clinical study. Method. 20patients with hemophilia A and B will be recruited in this study. Patients will be recruited in 3 regions of Spain. The dependent variables will be: bleeding frequency (self-registration), pain (measured with the visual analog scale), joint status (Hemophilia Joint Health Score scale), strength (dynamometer) and range of motion (goniometer). Three evaluations will be carried out: pre-treatment, post-treatment and after a follow-up period of 4 weeks. Expected results. Observe the safety of blood flow restriction in hemophilia patients. To analyze the efficacy of blood flow restriction in improving muscle strength, joint pain, range of motion, and joint damage in patients with hemophilic knee and ankle arthropathy.
This study will assess the safety and efficacy of multiple-dose of STSP-0601 for the treatment of bleeding episodes in hemophilia A or B patients with inhibitors.
The aim of the WFH GTR is to provide a database in which long-term data on PWH who receive gene therapy from around the world, will be collected and housed.
the aim of the SERAPHINE study is to gather data stemming from the French research database BERHLINGO (= Base d'Etude et de Recherche en Hémostase pour Les Investigateurs du Grand-Ouest, i.e. Database for Research on Hemostasis for the Investigators of Western France), in order to get a detailed report about the therapeutic management and use of turoctocog alfa (NovoEight®) in surgery in pwHA (Patient with inherited Hemophilia A).
This study will assess the pharmacokinetics and pharmacodynamics of STSP-0601 at five dose levels. The results will help identify the most optimal doses to treat bleedings in hemophilia patients with inhibitors.
This study programme aims to examine the real-world experience and impact of gene therapy in a diverse community of people and families affected by haemophilia who have been or will be exposed to gene therapy.