View clinical trials related to Hemophilia A.
Filter by:C3731003 is a pivotal Phase 3 study to evaluate the clinical efficacy and safety of a single IV infusion of PF-07055480 / giroctocogene fitelparvovec (Recombinant AAV2/6 Human Factor VIII Gene Therapy) in adult male participants with moderately severe or severe hemophilia A (FVIII:C≤1%) for the study duration of 5 years. The study will enroll eligible participants who have been followed on routine prophylaxis with FVIII products in the Lead-In study C0371004.
The purpose of this study is to assess the safety and effectiveness of Esperoct® for long-term routine use in patients with Haemophilia A. Participants will get Esperoct® as prescribed by their doctor. The study will last for about 2 years for each participant.
This Phase III clinical study will evaluate the safety and effectiveness of valoctocogene roxaparvovec in combination with prophylactic corticosteroids in patients with severe hemophilia A.
Uncertainty about how to treat existing hemophilic arthropathy (HA) is the most important issue that will increase functionality and joint health. Recent studies have shown that MT can be used safely for improving joint health, pain and ROM with no bleedings in PwH with elbow and ankle HA. Unlike MT studies in hemophilia, we investigated the effects of MT on important parameters such as muscle strength (MS), functional level, joint health, functional independence score in hemophilia (FISH) and kinesiophobia. These parameters are closely related to functional level and quality of life of PwH. Therefore, evaluation of these parameters and determining the effects of the MT on these parameters are very important for both PwH and also clinicians.
Manual treatment involves techniques based on the principles of moving the joint faces and separating the joint gap, and soft tissues mobilization techniques. The use of manual therapy in hemophilia is gradually increasing although it previously described as a contraindication. The utility and use of strengthening exercises in hemophilia has been going on for a long time. In this study, the effect of manual therapy and strengthening exercises joint was investigated in hemophilic arthropathy of the elbow.
In persons with severe haemophilia A (HA) infused factor VIII (FVIII) half-life and other pharmacokinetic parameters can vary according to determinants such as blood group, von Willebrand factor (VWF) level or age. However, FVIII pharmacokinetics (PK) has not been thoroughly studied in patients with severe HA as a function of daily physical activity. Patients with severe HA (FVIII <1%) are predisposed to prolonged bleeding following even minimal musculoskeletal injuries. Potential consequences of repeated musculoskeletal bleeding are pain, arthropathy and physical disability. The key standard of care for HA patients is prophylactic infusions of FVIII concentrates (25-50 IU kg-1 infused 2-3 x/week), depending on individual response. The level of infused FVIII decreases as a function of time according to both specific PK features of each product and biochemical/genetic characteristics of the patients or different clinical conditions. Some critical points remain still unraveled, for instance, whether or not FVIII AUC is significantly affected by physical activity/exercise, in response to increased metabolic rate or subclinical/microhaemorrhages in patients with severe HA. It is known that vigorous-intensity physical activity/exercise can transiently but significantly increase circulating levels of endogenous VWF and consequently FVIII in normal subjects and in patients with moderate or mild haemophilia A. The proposed study is a Proof of Concept one as it will be aimed at investigating the relation between daily physical activity, measured by SenseWear® armband device, as number of daily steps, and PK variability of infused rec-FVIII concentrate. This kind of investigation has never been done and it is a great interest also for the evaluation of patients' quality of life.
This is a multi-center randomized phase III clinical trial, the Inhibitor Eradication Trial, in which Eloctate ITI plus Emicizumab will be compared with Eloctate ITI alone to eradicate inhibitors in severe hemophilia A.
This is a multi-center randomized phase III clinical trial, the Inhibitor Prevention Trial, in which Eloctate will be compared with Emicizumab, using adaptive design, to prevent inhibitors in patients with severe hemophilia A.
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.
Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B. The current single country, multi-centric, open label, non-randomized clinical trial is a post-approval study to fulfill the Central Drugs Standard Control Organization (CDSCO) request for supplementary information relating to the use of nonacog alfa in Indian subjects with hemophilia B.