View clinical trials related to Sickle Cell Disease.
Filter by:Sickle cell disease is an inherited blood disorder that affects red blood cells (RBCs). People with sickle cell disease frequently experience anemia, or a low number of RBCs. RBCs are responsible for carrying oxygen to the brain and other body tissues that need oxygen to function properly. The purpose of this study is to determine what changes, which were possibly caused by anemia, exist in the brains of individuals with sickle cell disease.
This Phase 2a, multicenter, open-label, dose-escalation study is designed to assess the safety and biologic activity of daily oral administration of 4 escalating doses of sapropterin dihydrochloride over 16 weeks in subjects with sickle cell disease. During an optional extension phase, the study will assess the safety, tolerability, and efficacy of extended treatment with sapropterin dihydrochloride, for a total of up to 2 years; The extension phase of this study was terminated.
The study will be conducted at 15-20 US centers in a randomized, placebo-controlled, double-blind fashion. Enrollees will be hospitalized sickle cell disease (SCD) patients at-risk for acute chest syndrome (ACS) based on the presence of vaso-occlusive crisis (VOC), fever (T ≥38.0°C) and serum sPLA2 concentration ≥50 ng/mL.
This study will examine whether hypnosis can reduce the frequency and intensity of pain in patients with sickle cell disease. Patients 18 years of age and older with sickle cell disease and a history of pain associated with their disease may be eligible for this study. Participants are interviewed to assess their frequency and intensity of pain, sleep quality, coping strategies, mood and anxiety and are then randomly assigned to study Group A or B (see below). All participants are given pain diaries to complete at home and turn in at each clinic visit. They undergo the following procedures: Group A Weeks 1-4: Receive weekly 60-minute hypnosis sessions, in which they are given suggestions for relieving pain, reducing anxiety, improving sleep and enhancing their health and well-being. The sessions are audio- and videotaped. Week 5: Are interviewed to assess pain, sleep, coping strategies, mood and anxiety. Week 6: Receive a DVD player and DVD with instruction on how to perform self-hypnosis. They practice hypnosis at home as often as needed, but at least once a day. They record in a pain diary in the morning and the evening their amount of pain, medication use, school or work attendance, quality and amount of sleep and number of times they use self-hypnosis. Weeks 8, 10 and 12: Turn in their pain diaries and have a pain assessment. Week 12: Are assessed for how they respond to the hypnosis. Group B Weeks 1-4: Receive weekly 60-minute sessions of education about sickle cell disease. Week 5: Are interviewed to assess pain, sleep, coping strategies, mood and anxiety. Week 6: Turn in their daily pain diaries and receive a DVD player and DVD that contains educational materials about sickle cell disease. Weeks 8, 10 and 12: Turn in their pain diaries and have a pain assessment. Weeks 13-24: Follow the procedures described in weeks 1-12 for Group A.
Sickle cell disease (SCD) is a devastating chronic health condition that primarily affects African-Americans. Painful episodes are the most frequent form of morbidity in SCD and the most frequent reason for hospitalization. Cognitive-behavioral interventions for pain management have shown efficacy for improving coping abilities, reducing the amount of medication needed to manage pain, and improving daily functioning during painful episodes. However, difficulties with disseminating and implementing cognitive-behavioral treatments have resulted in almost no use of these techniques in pediatric settings. In South Carolina these difficulties are compounded by social and geographical factors that pose particular challenges. A major issue with implementing quality pain management protocols is the difficulty with providing adequate practice and monitoring of the use of the techniques, particularly given the rural population in South Carolina and transportation difficulties for economically disadvantaged families. Due to a history of under-treating pain in SCD it is also critical that psychological and medical treatments are presented in an integrated manner so that these approaches are viewed as complimentary, not mutually exclusive, approaches to pain management. Finally, we believe the same implementation issues for improving the use of behavioral coping skills are also important for improving adherence to medication protocols for appropriate home-based pain management. The purpose of this proposal is to develop, implement, and evaluate a pain management protocol that uses portable electronic devices and other technologies to increase the practice of psychosocial pain management techniques, improve adherence to the overall biopsychosocial pain management protocol, and improve the clinician's ability to track progress with fewer office visits. In addition to addressing important dissemination issues, by embedding methods to assess for adherence into the technology it will be possible to continuously evaluate and modify protocol efficacy, resulting in a product that is effective, empirically sound, and flexible. Participants will be randomly assigned to the intervention or waitlist control condition. Those on the waitlist condition will receive the same study procedures after a 2 month wait periods. We anticipate that the intervention will result in better pain management and less impairment in the participants.
This study will explore how people with sickle cell disease (SCD) develop a complication called pulmonary hypertension (PHTN), a serious disease in which blood pressure in the lungs is higher than normal. PHTN is also caused by HIV, hepatitis C and schistosomiasis. Patients who have both SCD and one of these other infections may develop more severe PHTN. The number of Nigerians with SCD who also have PHTN is not known, nor is the cause of PHTN in this population. This study will examine genetic material in people with and without SCD to determine whether certain genes will allow doctors to predict which patients with SCD are likely to develop PHTN. Nigerian males and females 10 years of age and older with or without SCD may be eligible for this study. Patients must have SS, SC, or SB thalassemia or other genotype; control subjects must have hemoglobin A or AS genotype. Participants undergo a complete medical history and physical examination, blood tests, electrocardiogram (EKG), ultrasound tests of the heart and abdomen, and a 6-minute walk (distance test) to determine exercise capacity. Blood tests include screening for HIV, hepatitis B and C, schistosomiasis, hookworm and malaria. Patients who test positive for HIV, hepatitis B or C, schistosomiasis, hookworm or malaria are referred for treatment at Ahmadu Bello University Teaching Hospital in Zaria, Nigeria, and those who test negative for hepatitis B are referred for vaccination. Genetic tests focus on genes involved in SCD, PHTN, inflammation, blood vessel function and red blood cell function.
The primary objectives of this prospective, observational study are (1) to describe the long-term cellular, molecular, and clinical effects of hydroxyurea therapy in sickle cell disease, and (2) to perform hydroxyurea pharmacokinetics studies. This study will follow sickle cell patients being treated with hydroxyurea for a long period of time to evaluate the long-term cellular and molecular effects of the drug on the patients' body. This study will consist of two patient groups. One group will be made up of patients who have received hydroxyurea therapy before entering the study. The second group will be made up of patients who have not received hydroxyurea before study entry.
This study will examine the use of hydroxyurea and erythropoietin for treating sickle cell disease in patients who also have kidney disease or pulmonary hypertension (high blood pressure in the lungs). Hydroxyurea increases production of fetal hemoglobin in the red blood cells of patients with sickle cell disease, reducing the amount of sickle cells that cause pain and other complications requiring hospitalizations. However, hydroxyurea treatment has limitations: patients with sickle cell disease who have developed kidney disease may not be able to get the full benefit of the medicine, and hydroxyurea alone may not be able to treat life-threatening complications such as pulmonary hypertension or stroke. This study will determine which of two dosing schedules of hydroxyurea and erythropoietin is more effective for treating patients with sickle cell disease who also have kidney disease or pulmonary hypertension, and will examine whether the two drugs can lower blood pressure in the lungs. Patients 18 years of age and older with sickle cell anemia and kidney disease or pulmonary hypertension, or both, may be eligible for this study. Candidates are screened with a medical history, physical examination, blood tests, a 6-minute walk test (test to see how far the subject can walk in 6 minutes), and echocardiogram (ultrasound of the heart to measure blood pressure in the lungs). Participants undergo the following tests and procedures: Stabilization Phase: Patients take 2 hydroxyurea tablets a day until their fetal hemoglobin levels stabilize, usually over 2 to 4 months. They have blood tests every 2 weeks to monitor hemoglobin and fetal hemoglobin levels. At some time during this period, they undergo a test to measure kidney function, in which they are injected with an iodine-containing dye and wear a small pump for 1 day that injects a small amount of dye under the skin over 24 hours. They come to the clinic for 2 or 3 blood tests collected over 4 hours. Sequence I (Standard): When the fetal hemoglobin levels have been stable for 2 months, patients have a repeat echocardiogram and 6-minute walk test. Erythropoietin is then added to the hydroxyurea regimen. It is given 3 days a week, as an injection under the skin, along with iron supplements. Patients have blood tests and blood pressure measurements every week or every other week. Patients with pulmonary hypertension have another echocardiogram and 6-minute walk test once the hemoglobin level is stable. Sequence II (Cycled): When hemoglobin levels have stabilized with hydroxyurea once a day and erythropoietin 3 times a week, the hydroxyurea is adjusted so that the amount taken in 7 days is "cycled" over 4 days, and the erythropoietin is cycled over 3 days, with the dose increased twice, every 3 to 4 weeks. Blood pressure and hemoglobin are monitored once or twice a month. Patients with pulmonary hypertension have another echocardiogram and 6-minute walk test once the hemoglobin level is stable. Patients who develop complications while taking the drugs have their treatment regimens adjusted as needed.
This is an open-label, non-randomized, multi-center trial designed to provide expanded access of deferasirox to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treated with locally approved iron chelators.
In this study, patient blood samples from NMA transplants will be provided by Pittsburgh, and samples from myeloablative transplants will be provided by Atlanta (comparative controls). Samples would be obtained pre- and post-BMT from the recipient at a total of 7 timepoints, and from the donor at one timepoint.