Active clinical trials for "Hemophilia A"

The purpose of this study is to see if giving azidothymidine (AZT) to HIV-positive patients with hemophilia is safe and if it is effective in lowering HIV levels and boosting the immune system. HIV infects and inactivates certain blood cells that are part of the body's immune system. The damage to the body's immune system can result in unusual infections and/or unusual forms of cancer. A large percentage of hemophiliacs are HIV-positive and there is a clear risk for the development of AIDS in these patients. AZT may be effective in lowering HIV levels and boosting the immune system but its side effects are not understood in these patients.

Hemophilia A and B are congenital, recessive X-linked disorders caused by lack or deficiency of clotting factor VIII (FVIII) or IX (FIX), respectively. The severity of the disease depends on the reduction of levels of FVIII or FIX, which are determined by the type of the causative mutation in the genes encoding the factors (F8 and F9, respectively). The hallmark clinical characteristic, especially in untreated severe forms, is bleeding (spontaneous or after trauma) into major joints such as ankles, knees and elbows, which can result in the development of arthropathy. Intracranial bleeds and bleeds into internal organs may be life-threatening. The median life expectancy was ~30 years until the 1960s, but improved understanding of the disorder and development of efficacious therapy based on prophylactic replacement of the missing factor has caused a paradigm shift, and today individuals with hemophilia can look forward to a virtually normal life expectancy and quality of life.

Nine Multiple Sclerosis (MS) patients suffering an acute relapse from the outpatient or inpatient settings will be consented to be followed prospectively for three months post relapse, in an effort to identify markers of incomplete relapse recovery. Factor VIII-related labs will be drawn for three months without influencing standard of care treatment decisions. During this time, patients will be followed with clinical and diagnostic assessments in addition to blood tests including: Expanded Disability Status Scale (EDSS), Multiple Sclerosis Functional Composite (MSFC), recovery surveys, and MRIs of the brain, cervical spine, and thoracic spine with and without contrast. Clinical, imaging, and Factor VIII-related lab data individually or in aggregate will be correlated with relapse presence, severity, and extent of recovery following standard treatment interventions.

This study prospectively investigates the safety, FVIII immunogenicity, and hemostatic efficacy of prophylactic HEMLIBRA® given with a concomitant low dose recombinant factor VIII (rFVIII) known as NUWIQ®, in HA infants and children <3 years old who have had little to no previous exposure to FVIII. In addition, the study investigates the safety and efficacy of a novel FVIII ITI regimen in children <21 with existing low and high titer inhibitors (LTI and HTI).

This study is an international, multicenter, open-label, single arm, prospective clinical trial and will evaluate the efficacy of prophylactic emicizumab administered on a scheduled basis to prevent bleeds in patients with acquired hemophilia A (AHA).

this study evaluates the effects of therapeutic exercises on kinesiophobia and health-related quality of life in adult haemophilia patients. half of participants will receive therapeutic exercises and verbal information about the positive effects of therapeutic exercises on physical pathologies due to hemophilic arthropathy while the other half will receive only verbal information.

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.

This is a research study of adults with hemophilia that have limitations in elbow joint motion that is the result of bleeding into the joint. People with hemophilia who have bleeding into their elbow joint may have limitations in elbow joint movement and pain in the joint. This research is being done to determine the effect a strength training program has on the amount of movement people with hemophilia and elbow joint disease have.

This study is focused on males who have Hemophilia B and who need regular preventive treatment with factor IX protein (FIX) replacement therapy to prevent and also to control their bleeding events. The aim of the study is to gather at least 6 months of information on bleeding events for each individual participant while they continue to use their usual FIX replacement therapy. There is no experimental treatment being tested in this study. The study is informational, and part of a larger program to understand and treat Hemophilia B with a potential experimental new therapy in the future. After you complete this research study, should you be interested and eligible, you may be offered the opportunity to take part in a future study with this new therapy. There is no obligation to agree to taking part in this future study. The study is looking to answer several other research questions to help understand each participant's individual disease characteristics, including: How often you use FIX replacement therapy, both on a regular basis (prophylaxis) and as needed to treat bleeding events Measurement of FIX activity (factor IX is a clotting factor) by different laboratories using different types of tests in Hemophilia B patients Possible complications from the FIX replacement therapy you receive (you will continue to use your usual standard of care FIX prophylaxis for Hemophilia B during the study) How your quality of life is affected by Hemophilia B How your joint health is affected by Hemophilia B How often you visit the emergency room, urgent care center, physician's office, hospital, or have a telemedicine visit as a result of bleeding events Whether the body makes antibodies (a protein produced by the body's immune system) against the FIX replacement therapy you receive, which could make the drug less effective or could lead to side effects

The main aims of the study are to learn if TAK-672 can control bleeds in participants with acquired hemophilia A and if the participants have side effects from TAK-672. Acquired hemophilia A is when people's immune system attacks specific proteins, known as clotting factors, in their bodies. This is different from hemophilia A, which is a condition people are born with. At the first visit, the study doctor will check who can take part. For those who can take part, participants will visit the clinic or hospital when they get their next bleed. They will receive TAK-672 slowly through a vein. This is called an infusion. They might need extra infusions of TAK-672 to control the bleed. After their bleed is controlled, participants will regularly visit the clinic for a check-up and to treat any further bleeds. This will happen until all participants have received their last dose of TAK-672 to control their 1st bleed. After this, all participants will visit the clinic 90 days later for a final check-up.