Active clinical trials for "Anemia, Sickle Cell"

The purpose of this study is to test the effects of a laboratory-based hypnosis session compared to an attention control condition on peripheral blood flow, autonomic stress responses, and acute pain responses in adolescents (ages 12-21) with sickle cell disease, and examine how perceived disease-related stigma may affect these responses.

This Phase 1 first-in-human, first-in-patient, single ascending dose and multiple dose study will be a randomized, double-blind, placebo-controlled investigation of the safety, tolerability, and pharmacokinetics of PF-07209326 in healthy participants and participants with sickle cell disease.

Background: Sickle cell disease (SCD) is an inherited blood disorder. It results from a single genetic change (mutation) in red blood cells (RBCs). RBCs are the cells that carry oxygen to the body. In people with SCD, some RBCs are abnormal and die early. This leaves a shortage of healthy RBCs. Researchers want to learn more about how long RBCs live in the human body. Objective: To study how long RBCs live in people with and without SCD. Eligibility: People age 18 and older who either have SCD, had SCD but were cured with a bone marrow transplant, have the sickle cell trait (SCT), or are a healthy volunteer without SCD or SCT Design: Participants will be screened with a medical history and physical exam. They will give a blood sample. Participants will have a small amount of blood drawn from a vein. In the laboratory, the blood will be mixed with a vitamin called biotin. Biotin sticks to the outside of RBCs without changing their function, shape, or overall lifetime. This process is known as biotin labeling of RBCs. The biotin labeled RBCs will be returned to the participant via vein injection. Participants will give frequent blood samples. Their RBCs will be studied to see how many biotin labeled RBCs remain over time. This shows how long the RBCs live. Participants will give blood samples until no biotin labeled RBCs can be detected. During the study visits, participants will report any major changes to their health. Participation lasts for up to 6 months.

Aim: Sickle cell disease (SCD) is a hereditary disease. Approximately 5% of the world's population carries trait genes for haemoglobin disorders, mainly, sickle-cell disease and thalassaemia. The incidence of sickle cell disease varies according to different geographical locations. Persons with SCD experience both acute and chronic pain. it is important that nonpharmacological therapies be investigated and used as complements to pharmacological therapies to address and treat both acute and chronic pain for those with SCD. An educational program involving parents with children with SCD can help parents use nonpharmacological methods for their children in reducing pain. The training program allows parents to develop their knowledge of the SCD and the importance of non-pharmacological methods, and may provide parents with the opportunity to develop preventive attitudes towards reducing pain crises. The research has two purposes. The first is to identify the non-pharmacological approaches that parents use to help their children with sickle cell disease in Turkey, Chad. Second, to determine the effectiveness of non-pharmacological approaches given to parents in Turkey, Chad. Method: This study is an experimental research method using a quasi-experimental design. This study used a pretest and posttest, with a design that is used is the two group pretest-posttest design. The study was conducted on the parents of children with sickle cell disease diagnosed and followed up in the Pediatric Hematology Oncology polyclinics of two hospitals in Chad in Central Africa between September 2015 and February 2016. The study found in April 2016 and July 2016 between Turkey's southern Antalya and Mersin two university hospital outpatient Children's pediatric hematology oncology has made clinic on sickle cell disease diagnosed and monitored the children's parents. The parents were selected using eligibility criteria and the study was performed in three steps (Pre-intervention testing session, education session, and post-intervention testing session). In the first step, "Information Form", "Parents' Experience of Nonpharmacological Methods Questionnaire", and "Nonpharmacological Approaches Used by Parents for Their Children and Knowledge of Parents about Nonpharmacological Approaches Questionnaire" questionnaires were applied to parents. In the second step an individual education was conducted by the researcher using the education book. In the third step, the questionnaire were reapplied after 3 weeks.

This is an open-label study to understand the safety and tolerability of AXA4010, a novel composition of amino acids in adult and adolescent subjects with sickle cell disease over 12 weeks. The study also assesses the effects of this amino acid composition on the structure and function of the vascular system. Physiological effects on structure and function will be assessed by Magnetic Resonance Imaging (MRI) to assess blood flow in the brain and kidneys and the 6-Minute walk with pulse oximetry. Changes in blood biomarkers of inflammation will also be assessed.

The Dissemination and Implementation of Stroke Prevention Looking at the Care Environment (DISPLACE) study is a multi-center, national, National Heart, Lung and Blood Institute (NHLBI)-funded grant to look at the real-world implementation of stroke prevention guidelines (STOP Protocol) in which transcranial Doppler (TCD), a measure of cerebral blood vessel velocity, is used to screen for stroke risk in children ages 2-16 with sickle cell anemia (SCA). Part 3 of the DISPLACE study is an implementation clinical trial designed to test novel implementation strategies with the goal of improving adherence and implementation of stroke screening. 16 of the lowest scoring implementation rates from DISPLACE Part 1 will participate in DISPLACE Part 3. All original 28 sites from DISPLACE Parts 1 and 2 will receive a patient and provider educational intervention including a re-branding of the TCD as "Sickle Stroke Screen" with a new infographic and educational materials. The 16 sites with moving to Part 3 will be provided a Provider reminder strategy, which is a web based application designed to remind providers of when patients are due for their Sickle Stroke Screen. These 16 sites will be randomized and 8 will be given an additional Patient Communication Strategy. These sites will have a single designed coordinator with whom patients will communicate with about scheduling, rescheduling, and any other questions regarding their Sickle Stroke Screen. Upon completion, data will be analyzed to compare those who have had TCD screenings done appropriately and those who did not as well as the overall effect of the multi level interventions on the changes in TCD rates.

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.

The purpose of this study is to measure the exposure to prasugrel's active metabolite and the pharmacodynamic effects of prasugrel treatment in people with Sickle Cell Disease (SCD).

An Open-label Study of GBT021601 in 8 to 10 healthy male or female participants to evaluate the absorption, distribution, metabolism, and excretion (ADME) of GBT021601.

Sickle cell disease (SCD) is a common genetic disorder characterized by episodes of pain, yet programs to assist SCD adolescents with better identification and communication about pain are lacking. Research shows that interactive gaming technology can enhance adolescents' learning, and can be especially effective in delivering health-related messages and tools to improve self-care. Pinpoint is an interactive gaming app that will be tested in a Phase II project to determine whether the app assists SCD teens with improving their communication and identification skills for pain self-report.