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NCT06818266
The purpose of this study is to determine whether a single infusion of tocilizumab is effective in reducing the time to successful weaning from both supplemental oxygen and any respiratory support, in pediatric and adult patients with sickle cell disease (SCD) during acute chest syndrome (ACS).
NCT04839354
The trial is designed to test intravenous (IV) arginine therapy in children with sickle cell disease (SCD) and vaso-occlusive painful episodes (VOE) to further knowledge on efficacy and safety of this orphan drug.
NCT07431398
This clinical trial is a study to evaluate the pharmacokinetics of the tablet formulation Pociredir in fasted and fed state participants with Sickle Cell Disease (SCD).
NCT06397118
The investigators are studying how to prevent malnutrition in children with sickle cell disease (SCD) in northern Nigeria. Community health workers will teach mothers about nutritious local foods that might help children grow better than the usual advice from doctors. The investigators will enroll 148 children with SCD aged 6 to 18 months old. The investigators will determine if their weight and diet improve and check for low vitamin A levels. This information will help us find better ways to improve growth for children with SCD.
NCT03958643
Background: Sickle cell disease is a common inherited blood disorder. Kidney disease is a major cause of problems in people with sickle cell disease. In order to identify kidney problems early and stop the progression of kidney disease, doctors need the most accurate tests to check kidney function. Researchers hope to understand more about how to test for kidney disease in people with sickle cell disease. Objective: To determine which of two different lab tests is the best to measure kidney function in adults with sickle cell disease. Eligibility: People 18 years and older who have sickle cell disease Design: Participants will be screened with a medical history and blood tests. Participants will have up to 3 visits. Participants will collect their urine in a special container over 24 hours. At the first visit, participants will have blood tests. They will bring their container of urine to the visit. They will have an iothalamate test. For the test, they will get a catheter: a small tube will be inserted into a vein. A special contract agent will be injected into the vein. Blood will be collected over the next 4 hours to test kidney function. Participants will return the next day for a second visit. They will have blood tests. They will have an MRI. For the MRI, they will like on a table that slides into a machine that takes pictures of the kidneys. They may have the MRI in a third visit. ...
NCT07616154
The purpose of this study it to evaluate a reduced toxicity conditioning regimen for haploidentical donor HCT followed by a GVHD prophylaxis regimen comprising of post-transplant cyclophosphamide, sirolimus and abatacept with the goal to improve the GVHD-free rejection-free survival (GRFS) to greater than 90% after haploidentical donor HCT in children and young adults with SCD. Primary Objective: \- To assess the GVHD-free and rejection free survival (GRFS) after haploidentical donor HCT in children and young adults with SCD. Secondary Objectives: * Assess the overall survival (OS) and disease-free survival (DFS) after haploidentical donor HCT for SCD. * Estimate incidence and severity of acute and chronic GVHD after haploidentical donor HCT for SCD. * Assess the neutrophil and platelet engraftment kinetics after haploidentical donor HCT for SCD.
NCT07543289
The wide interindividual variability in clinical response to hydroxyurea therapy in the management of sickle cell disease has limited its use. These variabilities have been linked to differences in pharmacodynamics, pharmacokinetics, and pharmacogenetics. This study, therefore, aims to enhance understanding of these factors as they relate to hydroxyurea therapy, with the overall goal of developing a precision medicine algorithm. The study will be a prospective cohort pharmacokinetic study of 100 Nigerian patients with sickle cell disease, including current hydroxyurea users and naive patients. Pharmacodynamic markers will be collected to evaluate response. PopPK and PK-PD models will be developed in Monolix, exposure-response relationships will be analysed in R, and pregnancy, lactation, and paediatrics PBPK models will be developed in Simcyp or PK-SIM to inform dose optimisation. This study aims to build a pharmacometric model by integrating differences in pharmacokinetics, pharmacodynamics, and pharmacogenetics of hydroxyurea, which could aid the optimisation of hydroxyurea for sickle cell patients in Nigeria. The objectives of the study are i. To determine the prevalence of genetic polymorphisms in metabolic enzymes and transporters relevant to the disposition of hydroxyurea in the Nigerian sickle cell disease population, ii. To develop and validate an analytical method for the quantification of hydroxyurea using high-performance liquid chromatography. iii. To evaluate the influence of genetic and other covariates on hydroxyurea disposition in the Nigerian sickle cell disease population using population pharmacokinetic modelling, iv. To investigate the relationship between hydroxyurea exposure and clinical outcomes (foetal haemoglobin, mean corpuscular volume, reduction in vaso-occlusive crises (VOC), and improved blood count) using pharmacokinetic-pharmacodynamic modelling, v. To develop physiologically-based pharmacokinetic (PBPK) models that could predict hydroxyurea concentrations in special populations of sickle cell disease patients in Nigeria i.e. pregnant women, lactating mothers, breastfed infants, and paediatrics. vi. To develop a dosing guideline for hydroxyurea therapy in Nigerian sickle cell patients. Overall, this study will provide scientific knowledge that can enhance clinical decision-making in sickle cell management within the Nigerian population, and the models could serve as a template to optimize hydroxyurea use in this population.
NCT06477289
This study will examine sleep disordered breathing and sleep quality in participants (ages 12-18) diagnosed with sickle cell disease of any genotype. We will utilize remote peripheral arterial tonometry (PAT) and questionnaires to evaluate difficulties with sleep. PAT assessments will occur remotely in the homes of participants. Neurocognitive, behavioral, and neuroimaging evaluations will occur on the same day as a routine clinic visit. Primary Objective: Evaluate the relationship between nocturnal oxyhemoglobin saturation (SpO2) and neurocognitive functioning (working memory and verbal comprehension) in children (ages 12-18) diagnosed with sickle cell disease controlling for age, genotype, and social vulnerability. Secondary Objective: Assess differences in white matter integrity, silent cerebral infarcts, neuroinflammation, and functional connectivity among children (ages 12-18) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age. Assess differences in self- and caregiver-reported mood and pain severity among children (ages 12-18) diagnosed with sickle cell disease with and without sleep disordered breathing after controlling for age. Exploratory Objectives: Explore the relationship between nocturnal oxyhemoglobin saturation (SpO2) and neurocognitive functioning (attention, processing speed, verbal memory, visual memory, motor dexterity) in children (ages 12-18) diagnosed with sickle cell disease controlling for age, genotype, and social vulnerability. Assess the feasibility of an ultraportable ring oximeter (BodimetricsCircul+ Ring) in children (ages 12-18) diagnosed with sickle cell disease. Assess the concordance between the Circul+Ring with the WatchPAT in children (ages 12-18) diagnosed with sickle cell disease.
NCT04610866
Background: Sickle cell disease (SCD) is a disorder that causes episodes of acute pain and progressive organ damage. Ways to manage SCD have evolved slowly. Treatments do not always work. Researchers want to see if a drug called mitapivat can help people with SCD. Objective: To test the long-term tolerability and safety of mitapivat (or AG-348) in people with SCD. Eligibility: Adults age 18-70 with SCD who took part in and benefited from NIH study #19H0097. Design: Participants will be screened with a medical history and physical exam. They will give a blood sample. They will have an electrocardiogram to test heart function. Participants will repeat some of the screening tests during the study. Participants will complete 6-minute walk tests to measure mobility and function. They will have transthoracic echocardiograms to measure heart and lung function. They will have dual-energy X-ray absorptiometry scans to measure bone health. They will complete online questionnaires that measure their overall health and well-being. Participants will take the study drug in the form of a tablet twice a day. Participants will keep a study diary. They will record any symptoms they may have. Participation will last for about 54 weeks. After 48 weeks, participants can either keep taking the study drug for 48 more weeks or be tapered off of the study drug to complete the study. Those who are on the study for 1 year will have 10 study visits. Those who are on the study for 2 years will have 14 study visits.
NCT06612268
This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can reduce the damage to different organs, improve your exercise tolerance and reduce fatigue in people with sickle cell disease.The participants will either get etavopivat or placebo. Which treatment the participants will get is decided by chance. Etavopivat is a new medicine and is currently being tested in other studies in addition to this one. The study will last for about 2 years.
NCT03708731
Despite the well-documented benefits of hydroxyurea (HU) therapy in decreasing morbidity and mortality in youth with Sickle cell disease (SCD), pediatric HU adherence rates range as low as 49% and lead to discontinuation of HU regimens in 8-20%. In addition, treatment non-adherence may lead to unnecessary increases in medication dosage resulting from erroneous assumption that a patient is non-responsive to treatment (versus non-adherent to the regimen as prescribed). Given the detrimental effects of non-adherence, assessment of and intervention for HU non-adherence is essential to improving health outcomes in the pediatric SCD population. Electronic adherence monitoring is widely considered the "gold standard" in objective adherence measurement. These monitors provide continuous, real- time records of medication adherence and reveal problematic behavior patterns, including underdosing, overdosing, delayed dosing, "drug holidays," and "white coat" adherence. Overall, electronic adherence measures are considered valid, reliable, and accurate, with clear advantages over pharmacy refill records, physician estimates and self-report measures. The primary purpose of this pilot study is to determine the use of the AdhereTech as a feasible and valid measure of HU adherence in pediatric SCD. Primary Objective Estimate the association between HU adherence as measured by the AdhereTech device to a) caregiver-report, b) youth-report, c) lab values, d) pill- count, and e) Medication Possession Ratio (MPR) adherence measures Secondary Objectives Estimate the rate of consent to the study, the rate of AdhereTech device use, the rate of AdhereTech device failure, and the perceived acceptability of using the AdhereTech device, as reported by caregivers and youth
NCT06820515
The Hemophilia Treatment Center (HTC) where you receive care is working with The American Thrombosis and Hemostasis Network (ATHN) to look at the quality of life of people with blood disorders and problems. Doctors, scientists, policymakers, and other health care providers need a large amount of information from a lot of people to answer scientific, public health, and policy questions about better ways to treat blood disorders. They will use the information from the ATHNdataset to answer these questions.
NCT07392216
This study aims to look at AMH levels in female children with SCD as they go through puberty to see if they are at the same level as other children without SCD at the same age and/or pubertal stage and will also look at how treatment exposures and pain crises affect the AMH levels in children with SCD. Primary Objective: * To evaluate whether AMH levels are lower in pre-teens and adolescent females with SCD when compared with healthy female controls (siblings, relatives, non-relatives of similar race/ethnicity) at the same age and pubertal stage. Secondary Objectives: * To evaluate whether AMH has a similar trajectory in female pre-teens and adolescents with SCD when compared with the general population and controls. * To describe pubertal timing, menstrual history, and markers of functional ovarian reserve (FOR), as well as prevalence of premature ovarian insufficiency (POI) as determined by medical history and laboratory markers in pre-teens and adolescents with SCD in comparison with their female controls. * To correlate AMH levels with FSH and estradiol levels, normal pubertal timing, and menstrual history in children and adolescents with SCD. * To correlate the severity of SCD (number of vaso-occlusive events) with pubertal timing, presence of normal vs abnormal menstruation, and laboratory markers of FOR, in pre-teens and adolescents with SCD. * To correlate the use of SCD modifying treatment modalities with pubertal timing, menstrual pattern, and laboratory markers of FOR in pre-teens and adolescents with SCD.
NCT07247188
The purpose of this study is to measure whether PCV21 vaccine (investigational pneumococcal vaccine) is safe and can help the body to develop germ-fighting agents called "antibodies" (immunogenicity) compared with 20vPCV (licensed pneumococcal vaccine) when given as a single dose to children aged 2 to 17 years with sickle cell disease who had received or not a previous vaccination with pneumococcal conjugate or pneumococcal polysaccharide vaccine.
NCT03720626
The purpose of this registration is to list Managed Access Programs (MAPs) related to SEG101, Crizanlizumab
NCT07064174
Kidney disease is a major cause of illness and death in people with sickle cell disease and sickle cell trait. Despite these concerning facts, we do not (1) have an in-depth understanding of how kidney disease starts in sickle cell disease and sickle cell trait, (2) have detailed insights into why kidney disease is worse in people with sickle cell disease and sickle cell trait, (3) have management options that are tailored to treating or preventing kidney disease in people with sickle cell disease or sickle cell trait. The SCeK Biorepository is a specialized, secure repository designed for the collection of blood and urine samples from people with sickle cell disease and sickle cell trait. These samples are connected to detailed medical records, with the sole purpose of allowing researchers to better understand how kidney disease starts and progresses in people with the sickle cell gene. By studying these stored samples (using new tests) together with health information, researchers can find better early warning signs of kidney injury and develop better ways to protect kidney health in people with sickle cell disease and sickle cell trait.
NCT05736419
Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.
NCT05640271
The investigators are evaluating the role of a low dose of tocilizumab in treating acute chest syndrome in patients with sickle cell disease. Tocilizumab inhibits interleukin-6 (IL-6) receptors and is used to treat rheumatoid arthritis and severe cytokine release syndrome, which can be seen with chimeric antigen receptor T-cell (CAR-T) therapy, and it is also authorized for treatment of COVID-19. Since IL-6 levels are elevated in the sputum of patients with acute chest syndrome, the investigators are hopeful that this will be an effective strategy. The investigators will be looking at how a low dose of tocilizumab affects oxygen status, clinical outcomes, and laboratory markers in patients admitted to the hospital with acute chest syndrome.
NCT06511453
The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupressure analgesia in patients with sickle cell disease (SCD).
NCT07187973
This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.