View clinical trials related to Hemophilia A.
Filter by:This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase. The IMP AMT-061 is a recombinant adeno-associated viral vector of serotype 5 (AAV5) containing the Padua variant of a codon-optimized human FIX complementary deoxyribonucleic acid (cDNA) under the control of a liver-specific promoter. The IMP is identified as AAV5-hFIXco-Padua (AMT- 061). The pharmaceutical form of AMT-061 is a solution for intravenous infusion. The administered dose of AMT-061 will be 2 x 10^13 gc/kg.
Repeated hemarthroses in patients with hemophilia may lead to hemophilic arthropathy with marked inflammation and synovial hypertrophy. Power Doppler ultrasonography is a useful tool in hemophilic arthropathy for assessment of disease activity and for monitoring response to treatment. Imaging inflammation with glucose analogue fluorine-18-fluorodeoxyglucose positron emission tomography (18F-FDG PET) is based on that infiltrated granulocytes and tissue macrophages use glucose as an energy source. Metabolism and 18F-FDG uptake increase when inflammation occurring. The purpose of this study is to investigate the associations between 18F-FDG PET/CT and Power Doppler assessment in patients with hemophilic arthropathy.
The primary objective of this study is to assess the pharmacokinetics in patients with severe hemophilia. The secondary objective is to assess the pharmacodynamics of BAY1093884 based on tissue factor pathway inhibitor activity
This study will test the well-known medicine turoctocog alfa for any side effects. The purpose is to test turoctocog alfa for any side effects in the Indian population. The participants will get turoctocog alfa. Turoctocog alfa is already a well-known medicine in India, and can be prescribed by the study doctor. The participants will get an injection every second day or 3 times per week. This is decided by the study doctor. The study doctor will decide the amount and how often the participants must take the medicine. The study will last for about 16 weeks. The participants will have 5 visits with the study doctor. If the participants agree to participate in this study, the participants will receive the first injection at the second visit, thereafter the participants will be trained to do the injection by themself.
This long-term follow-up study will continue to evaluate the long-term safety and efficacy of SPK-8011 and SPK-8016 in males with hemophilia A, who have received a single intravenous administration of SPK-8011 or SPK-8016 in any Spark-sponsored SPK-8011 or SPK-8016 study.
An online web-based cross-sectional survey in which participants will complete a distinct questionnaire for patients with hemophilia and obesity (PwHO) and/or their carers/spouses/partners, or a distinct questionnaire for healthcare professionals. Each questionnaire consists of more than 100 items including pre-coded (multiple choice) closed-ended questions, yes/no questions and rating scales. It is estimated that this one-off survey will take approximately 45-60 minutes to complete. As a cross-sectional study, there will be no treatment of patients.
Primary Objective: -To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by the frequency of bleeding episodes. Secondary Objectives: - To evaluate the efficacy of fitusiran compared to on-demand treatment with factor concentrates, as determined by: - The frequency of spontaneous bleeding episodes. - The frequency of joint bleeding episodes. - Health-related quality of life (HRQOL) in participants >=17 years of age. - To determine the frequency of bleeding episodes during the onset period. - To determine the safety and tolerability of fitusiran.
The purpose of this study was to determine the frequency of bleeding episodes in participants receiving fitusiran as prophylactic treatment of hemophilia compared to participants who were assigned to continue with their regular medication. In addition, the study assessed safety, quality of life, pharmacodynamics (PD), and pharmacokinetics (PK).
Phase 2, multi-center, open-label study designed to evaluate the PK, bioavailability, PD, efficacy and safety of a daily subcutaneous [SC] treatment regimen with MarzAA for bleeding prophylaxis in 12 adult subjects with hemophilia A or B with an inhibitor and history of frequent spontaneous bleeding episodes.
This US study will assess hemophilia A patient characteristics, health history and reasons for switching or not switching from both patient/caregiver and physician perspectives. For this purpose, this research study will include hemophilia A: 1) patients who have switched from conventional therapy to new FVIII products with an improved PK profile. 2) patients who remain on conventional therapy (who have never switched) but have considered switching, including those patients who switched from conventional therapy to new FVIII products with improved pharmacokinetics and then subsequently "switched back" to conventional replacement therapy. In doing so, real world evidence will be obtained from both patient and physician perspectives offering key insights for effective therapeutic management of patients with hemophilia A and to more fully understand what drives patient switching from a patient perspective and a physician perspective.