View clinical trials related to Anemia, Sickle Cell.
Filter by:Sickle cell disease (SCD) is one of the most common inherited diseases worldwide and exhibits highest frequency in people of African descent. Patients with SCD currently have few treatment options, with hydroxyurea being the only medication approved to reduce the frequency of vaso-occlusive crisis (VOC) and prevent other SCD complications such as acute chest syndrome. Once patients develop VOC, hospitalizations aim to alleviate pain; no specific therapy is currently available to otherwise affect the course of the VOC. However, there has been increasing interest in the role of coagulation in the pathogenesis of SCD. The investigators hypothesize that low dose anticoagulant therapy, such as prophylactic dose low-molecular-weight heparin (LMWH), could be a novel way to ameliorate the vaso-occlusive process and thereby hasten the resolution of pain.
The goal of this research study is to establish chimerism and avoid graft-versus-host disease in patients with hemoglobinopathies.
The purpose of this study is to assess whether methylphenidate is effective in enhancing the cognitive performance of children with the HbSS or HbSC genotype of SCD who have sustained neurological complications on laboratory-based measures of sustained attention, reaction time, and executive functions, and indirectly, verbal short-term and long-term memory.
Background: - Sickle cell disease often causes crises, with episodes of pain. Many people with sickle cell disease also have pain between crises. Inflammation is an important part of sickle cell pain. It may be related to levels of nitric oxide. Nitric oxide is a gas in the body that helps relax blood vessels and may be related to the pain from sickle cell disease. Researchers want to study the relationship between blood levels of nitric oxide and pain in people with sickle cell disease. Researchers also want to study how certain genes express themselves related to sickle cell pain. Objectives: - To collect blood samples and other genetic expression information to study sickle cell pain and its relation to nitric oxide levels in the blood. Eligibility: - People at least 18 years of age who have sickle cell disease. - Healthy volunteers at least 18 years of age. Design: - This study requires a screening visit and four study visits scheduled 1 week apart. Each visit will last about 1 hour. - Participants will be screened with a medical history and physical exam. They will complete questionnaires about pain levels (if any). They will also provide blood samples for genetic and other testing. - Participants will have a breath test to see how much nitric oxide they exhale. They will also have a test of their ability to detect small changes in temperature and touch. - Participants will keep a diary to record daily pain levels and pain medicines taken. They will write down what they eat to track foods that contain nitrates (such as meats like ham and bacon and vegetables like beets and spinach). - At each of the four study visit, participants will bring the pain diary, provide blood samples, and have breath nitric oxide tests.
This is a pilot study of hydroxyurea versus placebo to reduce central nervous system complications (abnormally fast blood flow to the brain, silent cerebral infarct or stroke) in young children with sickle cell disease. The investigators plan to identify children 12 to 48 months old without central nervous system complications and randomly assign 20 to treatment with hydroxyurea and 20 to treatment with placebo for 36 months. Neither the study doctors nor the participants will know which treatment they are receiving.
The pathophysiology of sickle cell disease (SCD) manifestations, are complex with interactions of intracellular hemoglobin, membrane and endothelial activation but the hallmark remains recurrent and painful vaso-occlusive episodes (VOC). These painful episodes are thought to result from ischemia caused when small blood vessels are occluded by misshapen, inflexible erythrocytes. Painful episodes are the most common cause of hospitalization, morbidity, and impairment for SCD patients. There is no therapy that completely prevents or directly aborts painful events for all patients. Consequently, treatment for acute VOC is primarily supportive using hydration and medicinal pain control. Every pain medication has the potential to relieve pain but is associated with significant limitations and side effects. The primary hypothesis to be tested in this double blind, randomized controlled trial is that Nalbuphine is equivalent to morphine for pain control and patients will suffer fewer episodes of acute chest syndrome. The investigators also expect subjects will report fewer side effects from respiratory depression, abdominal distention from reduced peristalsis, reduced histamine release causing pruritis and still be provided adequate pain control. Further hypotheses to be tested is ability to recruit patient participants while being treated in the Emergency Department and that continuous infusion of Nalbuphine with accompanying patient controlled analgesia (PCA) is safe and effective in controlling pain, requiring less total opiates consumption, while decreasing length of hospitalization.
Background: - In sickle cell disease (SCD), the proteins in the red blood cells that carry oxygen do not behave normally. In parts of the body where there are low levels of oxygen or where oxygen is used more, the sickle hemoglobin proteins may change shape and stick together. This causes the red cells to clump, which reduces blood flow. This leads to even lower oxygen levels and causes damage and/or pain. - One way to stop the red blood cells from sticking together is to increase the levels of fetal (baby or good ) hemoglobin. The good hemoglobin then takes the place of the sickle hemoglobin. - Hydroxyurea is the only approved drug for SCD. But hydroxyurea works in only about two-thirds of people with SCD. Even in those cases it sometimes stops working over time. - Researchers are interested in testing decitabine. The drug may help to increase fetal hemoglobin levels. But it has not yet been approved to treat SCD. Objectives: - To test the safety and effectiveness of decitabine in increasing fetal hemoglobin levels and improving the symptoms of sickle cell disease. Eligibility: - People at least 18 years of age who have sickle cell disease that has not improved after at least 6 months of hydroxyurea therapy. Those who cannot take hydroxyurea because of side effects may also participate. Design: - Participants will be screened with a physical exam and medical history. They will also have blood and urine tests, a lung function test, and other tests as required. - Participants will receive decitabine injections up to twice a week for 1 year. Depending on the response to treatments, the dose will remain the same or be reduced to once a week. - Participants will be monitored with frequent blood tests and other studies as directed by the study doctors. - After the study is completed, participants will go back to their usual sickle cell care. If decitabine has improved a participant's SCD, treatment may be continued under regular health coverage insurance if this can be arranged.
Prolong proposes to test safety, tolerability and pharmacokinetics of SANGUINATE™ in sickle cell disease (SCD) patients. Prolong's preclinical studies showed that SANGUINATE™ was safe in a number of different animal models and toxicology studies. In this Phase I trial, Prolong will test whether it is also safe and tolerable in sickle cell patients. The study will be conducted in 15 adult (>18 years) patients.
The research proposed is a pilot study of pediatric and adolescent/young adult patients who have received the curative intervention (MSD-SCT), disease-modifying interventions (HU or CT) or SCC (control), with respect to three clinically important outcomes: quality-of-life (QOL), neurocognitive function, and reproductive potential. Comparable cohorts will be identified for each of the groups, drawing from patients treated by the SCD program of Children's Healthcare of Atlanta (CHOA). QOL measures and neuropsychiatric testing and will be administered. Reproductive endocrine function markers (laboratory studies and pubertal staging), will be collected and analyzed. A tracking system of such patients will also be developed, gathering available retrospective data and setting up a mechanism for collection of new data.
Sickle Cell Anemia is caused by an inherited hemoglobin disorder. Healthy red blood cells are discoid and can deform and move through small blood vessels to carry oxygen to all parts of the body. In sickle cell disease, as red blood cells circulate and oxygen is released in the circulatory system, the deoxygenated abnormal hemoglobin S can begin to polymerize. When this occurs, the red blood cells can become sticky and elongated. These sickled red blood cells are less flexible and will obstruct small blood vessels and block normal red blood cells from traveling through the circulatory system, which limits oxygen delivery to tissues and organs. This is known as a "sickle crisis". Patients suffering from a sickle crisis experience severe pain and are at risk of stroke, heart attack or even death. By lowering the level of oxygen pressure at which sickling occurs and opening the vasculature and rapidly delivering oxygen directly to ischemic tissues, the addition of MP4CO to existing treatment protocols may alleviate pain associated with a sickle cell crisis, abort a crisis and/or potentially reduce the duration of a crisis. This could mean less time in the hospital and an improved quality of life for patients with sickle cell anemia.