View clinical trials related to Hemophilia A.
Filter by:This trial is conducted in Africa, Asia, Europe, Japan, and North and South America. The aim of this trial is to evaluate the safety and efficacy of activated recombinant human factor VII analogue (vatreptocog alfa (activated)) in haemophilia patients with inhibitors.
To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including: 1) Unknown adverse reactions, especially serious adverse reactions; 2) Changes in the incidences of adverse reactions under the routine drug uses. 3) Factors that may affect the safety of the drug 4) Factors that may affect the effectiveness of the drug
The purpose of the study is to compare the effects of recombinant factor VIIa (NovoSeven) on restoring coagulation activation between patients with hemophilia A and antibodies to factor VIII and normal subjects (controls) by use of an in vivo method.
This study is designed to evaluate BeneFIX infused as prophylaxis regimens, compared with BeneFIX administered as an on-demand regimen only. In the trial, subjects will participate in 4 study periods. The first period is a 16-week on-demand treatment, during which subjects will utilize BeneFIX to treat bleeding events episodically only as they occur (i.e., on-demand treatment). At the end of this period, subjects will be randomly assigned to 1 of 2 BeneFIX prophylaxis regimens: either 100 IU/kg once weekly or 50 IU/kg twice weekly, and followed for 16 weeks. At the end of this period, subjects will use on-demand treatment for 8 weeks. The purpose of this 8-week period is to mitigate the potential carry-over effect of the prior prophylaxis regimen on bleeding frequency. Following this 8-week on-demand treatment, subjects will cross-over and receive the alternate study prophylactic regimen for 16 weeks. The primary endpoint is annualized number of bleeding episodes, compared between the first on-demand period and each of the prophylaxis regimens. A comparison of annualized bleeding episodes between the two prophylaxis regimens will also be performed. Subject-reported outcomes will also be collected using a patient diary. At 24 and 48 hours following the onset of each joint bleeding episode, information on pain, sleep, and work will be collected. Additional data on physical functioning will be recorded on the diary at the end of the 16-week on-demand period, and at the end of each prophylaxis period. Information on safety will also be collected. Each subject will participate in this study for approximately 59 weeks (15 months), including a screening period of up to 3 weeks, an initial 16-week on-demand period, two prophylaxis treatment periods of 16 weeks each, separated by an 8-week on-demand treatment period. A modified FIX recovery study will be performed once during each prophylaxis period. The study diary will also be used by subjects to collect secondary endpoints. These endpoints, which are subject-reported outcomes, will be recorded in the diary at 24 and 48 hours following the onset of each joint bleeding episode. Additional data on physical functioning will be recorded on the diary at the end of the 16-week on-demand period, and at the end of each prophylaxis period. Patients will be recruited in the United States, Canada, Europe and Russia.
The purpose of this study is to compare the hemostatic efficacy and safety of continuous infusion versus intermittent bolus infusion in the peri- and post-operative setting, employing rAHF-PFM, a recombinant antihemophilic factor manufactured without added human or animal proteins, in previously treated patients with severe or moderately severe hemophilia A (baseline factor VIII level <= 2% of normal) who are undergoing unilateral major orthopedic surgery that requires drain placement. The total study period per subject (from consent to study completion) will vary from approximately 9 to 26 weeks and will involve clinical and laboratory assessments.
This international study will identify genetic factors that may influence the development of inhibitory antibodies in patients with hemophilia A after treatment with factor VIII. Bleeding episodes in patients with inhibitors are often more difficult to treat. Previous research indicates that genetic factors play a role in the development of inhibitors. A better understanding of the influence of genes in this treatment complication may be helpful in predicting, treating or preventing inhibitors. People in families in which one or more members have severe factor VIII deficiency and one or more have a history of an inhibitor may be eligible for this study. Participants fill out a form with questions about the person's relationship to other family members taking part in the study. Those with hemophilia provide a brief medical history, including hemophilia-related information, inhibitor history and the presence of other conditions such as hepatitis C and HIV. All participants have a blood sample taken for laboratory and research tests.
The Second Multicenter Hemophilia Cohort Study (MHCS-II) will evaluate and prospectively follow approximately 4500 persons with hemophilia who were exposed to hepatitis C virus (HCV). The vast majority will have been infected with HCV, and approximately 1/3 will have been infected with human immunodeficiency virus (HIV). Primary objectives are to quantify the rates of liver decompensation, hepatocellular carcinoma, and non-Hodgkin lymphoma and to evaluate candidate clinical, genetic, virologic, serologic and immunologic markers that are likely to be on the causal pathway for these conditions. Candidate clinical and laboratory markers will be examined longitudinally to define changes over time and their relationships to one another. Collaborative studies will focus on genome scanning and evaluation of candidate genetic loci for susceptibility or resistance to HCV and HIV infections or to the diseases that result from these infections. Additional studies will identify response and complication rates of various anti-HCV and anti-HIV regimens in the setting of comprehensive clinical care of persons with hemophilia.
Background: Over 80% of the hemophiliac population who became infected with HIV prior to 1985 are also co-infected with HCV. Thus, hemophiliacs represent an important population for studies of the natural history of these chronic viral infections. Moreover, the high rate of co-infection makes it an ideal group for assessing the interaction between the viruses and the relationship between viral specific immune responses and clinical progression. Although the hemophiliac poulation is unique, co-infection by these chronic viral pathogens is becoming increasingly common, particularly amongst intravenous drug users, who account for approximately 25% of the HIV-1 epidemic in the United States. Objectives: The aim of this study is to determine if polymorphism in the promoter region of TH1 and Th2 cytokines are associated with (1) intracellular cytokines levels in CD4 + Tcells, (2) Human Immunodeficiency Virus (HIV) and Hepatitis C virus (HCV) viral loads, and (3) clinical progression of HIV1 to AIDS in hemophiliacs. Eligibility: The current proposal will investigate host genetic factors related to HIV-1 and HCV immunopathogenesis by studying children and adolescents enrolled in the Hemophilia Growth and Development Study (HGDS). Design: This study is in collaboration with the principle investigators of the Hemophilia Growth and Development Study (HGDS) as part of a grant "Pathogenesis of HIV and HCV in Hemophilia: HGDS-3" with funding support by NIH/NICHD for the period 9/25/01 through 8/31/2005. This multicenter, United States study represents a well-characterized, prospectively followed cohort of HCV-infected hemophiliacs, of whom 207 are HIV-1 co-infected. Enrollment of the hemophiliac cohort was completed between 3/89 and 6/90. The final observation of the cohort (follow-up 16) was concluded during 7/98. No new samples or clinical data will be collected on this population. The LGD plays two roles in this project: (1) an administrative role overseeing the withdrawal, handling, and transport of samples from the HGDS/LGD and central repositories at the NCI-Frederick, and (2) a scientific role continuing investigations to determine the role of host genetic factors in Th1 and Th2 immune response and regulation of HCV and HIV viral replication..
Hemophilia A is a serious blood clotting disorder caused by a lack of factor VIII, a specialized protein needed for normal blood clotting to occur. Individuals with this disease may experience spontaneous bleeding, pain and swelling in their joints due to excess bleeding, and bruising. A common treatment for severe hemophilia A is to intravenously replace the deficient blood clotting factor; however, some individuals may develop antibodies to this replacement factor. This study will evaluate the effectiveness of rituximab at reducing the antibodies that develop in response to the replacement factor in individuals with severe hemophilia A.
Hemophilia, which results from deficiency of factor VIII or IX, is a common hereditary X-linked bleeding disorder affecting up to 10/100,000 population. About 60-70% of them have severe disease (factor level <1%). This group is characterized by the occurrence of frequent spontaneous bleeding into joints and soft tissues. If inadequately treated, it results in progressive damage to joints and muscles leading to crippling deformities. Close clinical observation of these patients over many years has shown that those with >1% levels have much less bleeding compared to those with less than 1%. This observation has gained immense clinical importance in planning therapy for these patients. To prevent progressive joint damage, the missing factor needs to be replaced. Much has evolved in this practice in the last 50 years. From administration of whole blood in the beginning, to plasma and cryoprecipitate, to purified plasma-derived concentrates and finally recombinant factor concentrates. The standard of therapy now is to replace factors frequently enough to maintain >1% factor levels at all times (“prophylaxis”) or administer immediately on premonition or earliest signs of bleeding (“on demand” therapy). This has greatly enhanced the quality of life of people with hemophilia. However, the optimal regimens of factor replacement remain to be defined. The definition of what is optimal management of this chronic condition, currently incurable for the vast majority of patients, varies significantly in different parts of the world, depending on practicality and social expectations. Models have care have been developed in Western countries based on careful documentation of outcome over many years. Such data is lacking from developing countries. This multi-center study aims to systematically record the outcome of musculoskeletal function in people with hemophilia in developing countries for the first time and provide information that can help plan care for the 80% of all hemophiliacs in the world who live in these countries. Currently there is no well documented model of care at the range of factor replacement practiced in these countries nor is there any significant information on the long-term outcome of musculo-skeletal function among these patients.