View clinical trials related to Sickle Cell Disease.
Filter by:Background: Peripheral blood stem cell transplantation procedures are used for people with sickle cell disease. Researchers want to improve the success and reduce the complications for these procedures. This might allow more people to have a transplant. Objective: To see if a new transplant regime is effective, safe and well tolerated in people with sickle cell disease. Eligibility: Adults at least 18 years old with sickle cell disease and certain complications. A relative who is a half tissue match. Design: Participants will be screened with medical history, physical exam, and blood tests. Recipients will also have: - Heart, lung, and mental health tests - Chest x-rays - Bone marrow taken from the pelvic bone - Eyes and teeth checked Recipients will have a large central line inserted into a vein for up to 6 months. Donors will have their veins tested and have an IV inserted for 1 day or on rare occasions 2 days. Donors will get a drug to activate bone marrow. It will be injected for about 6 days. Donors will have at least 1 five-hour procedure where bone marrow stem cells will be collected. Blood will be taken from a vein in one arm or in rare cases from a groin vein and put through a machine. Some blood will be saved and the rest will be returned. Stem cells will be taken from the saved blood in a lab and frozen until ready to give to the recipient. Recipients will have: - Stems cells collected and frozen - Hygiene lessons - Bone density scans - Low-dose radiation - Drugs for their immune system - Donor cells infused through their central line - Transfusions After about 30 days, recipients will leave the hospital. They must stay near NIH for 3 months after the transplant and have frequent visits. After returning home, they will have 8 visits over 5 years, then be contacted yearly. ...
Background: Sickle Cell Disease (SCD) is a blood disorder that occurs mainly in people of African descent. Researchers want to learn more about the painful attacks and complications associated with SCD. They want to look for a relationship between SCD and specific changes in the blood. They want to study the role of genetics, inflammation, and blood clotting factors in SCD. They will do this with blood samples collected during an acute painful attack and in between attacks. Objective: To learn more about the painful attacks and complications associated with SCD. Eligibility: People ages 18-80 with SCD or who are healthy Africans or African Americans without SCD Design: - Participants will be screened with medical history and physical exam. - Healthy participants will have one visit. - Participants with SCD will have their first visit when they are not having a pain attack. They will have their next visit during a pain attack. About 3-4 months after this attack, they will have a final visit. - Visits will include a physical exam, and blood and urine tests. - Participants may have their blood samples used for genetic testing for research.
This is an open label, single arm study which enrolled 5 subjects with SCD who previously participated in the GBT440-001 study (NCT02285088).
The 6-minute walk (6MWT) test is used in adults and children affected by a wide range of chronic diseases to evaluate their sub-maximal exercise capacity. It reflects the global response of various physiological systems (respiratory, cardio-vascular, neurologic, metabolic and musculosquelettic) in a situation simulating a daily life activity. In children with sickle cell disease, the 6MWT is correlated with a low level of hemoglobin, a low level of fetal hemoglobin and low red cell deformability. Our team previously reported that in a population of children with sickle cell disease, highly treated with hydroxyurea, the sole factor which was independently linked to the 6MWT was the presence of silence infarct. As the cardio-vascular and cerebro-vascular injury in sickle cell disease are directly correlated with hemolysis, the investigators aim to evaluate a) the clinical relevance of endothelial and inflammation parameters and new hemolysis markers and b) if the presence of silent infarct and the 6MWT are correlated with this biological markers. This cross-sectional study will include sickle cell disease patients regularly followed for more than 5 years at Hôpital Universitaire des Enfants Reine Fabiola, Centre Hospitalier Universitaire (CHU)-Brugmann, Centre Hospitalier Etterbeek-Ixelles, CHU Saint-Pierre, Cliniques Universitaires Saint-Luc (Bruxelles, Belgium). Inclusion criteria are: sickle cell disease (SS, Sbeta°, SC, Sbeta+), age range : 6 to 25 years, signed informed consent. Exclusion criteria are: transplanted patients, inability to perform the 6MWT (severe cognitive disability, femoral osteonecrosis with functional impairment), hospitalization and/ or transfusion in the last 3 months for acute event. Demographic data and clinical data will be retrospectively recorded. Blood test and 6MWT will be performed in steady state. Studied analysis will be: coagulation factors, free hemoglobin, Pro-B type natriuretic peptide (Pro-BNP), High sensitivity C reactive protein (HS-CRP), Intercellular Adhesion Molecule (ICAM), Vascular Cell Adhesion Molecule (VCAM) and Selectins. With this study, the investigators expect to validate new predictive markers for cardio-vascular or cerebrovascular injury and to identify patients at high risk to develop these complications.
The overall goal of this proposed project is to 1) increase co-management between sickle cell specialists and primary care providers (PCP's); 2) increase the use of hydroxyurea (HU) which prevents Vaso-Occlusive Episode (VOE), EDs and subsequent hospitalizations, and death; 3) identify and link patients not receiving primary or SCD specialty care to care, and 4) shift healthcare use from EDs and hospitalizations to primary and specialty co-management. Many persons with SCD experience a poor quality of life, serious medical complications and frequent painful events that require treatment from SCD specialty care, primary care and emergency department (ED) providers. There are two dominating models of care in the United States; neither are ideal. Many people with SCD have all of their healthcare needs addressed by sickle cell specialists who do not typically provide primary care and are often geographically distant from the patients' home. Other sickle cell patients receive all of their care in EDs. Both models are inadequate and result in an alarmingly high number of ED visits for many patients. Current care models are neither cost efficient nor promoting optimal patient outcomes. To improve outcomes, the investigators will implement a new model of care for SCD using nurse care managers, web based-interactive algorithms, and test if additional patient provided coaching can improve outcomes.
A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease
The primary aim of this study is to test the feasibility and acceptability of implementing a multimedia computerized cognitive behavioral therapy (cCBT) program for reducing SCD pain symptoms in a single-arm pilot pragmatic clinical trial. The investigators will recruit 40 SCD patients with chronic pain and/or on chronic opioid pain treatment and randomize them 3:1 to two groups (cCBT and e-Education respectively), randomizing unevenly in order to best gather feasibility data for the cCBT. Both groups will use a mobile app to track daily pain/mood. The cCBT group will receive sessions of the CALM-SCD program to complete via mobile device and will have weekly follow-up with a care coach. The Education group will receive online education modules to complete via mobile device and will also have weekly follow-up with a care coach. The primary outcomes of the trial include feasibility (recruitment, retention, provider and patient feedback) and acceptability (sessions completed) of the CALM-SCD program.
The investigators propose to adopt sustainable community networks (in this case churches) to implement an integrated community-based screening that incorporates mobile health technology (mHealth) to make prenatal test results available at the point-of-delivery to guide medical management.
This Phase 1b study in adults with sickle cell disease (SCD) in steady-state (non-acutely ill) aims to evaluate safety and toxicity of NVX-508 in a multi-dosing paradigm as well as to determine the maximum tolerated dose (MTD) in this population. The information gained from this study will be used in making decisions about the appropriate dose(s) and dosing schedule in future multicenter studies of the efficacy of NVX-508 in the treatment of vaso-occlusive episodes (VOE).
There are approximately 90,000 individuals in the United States with sickle cell disease (SCD). Studies have shown that up to 98 percent of patients with Sickle Cell Disease have a vitamin D deficiency, defined as a 25-hydroxyvitamin D level (25(OH)D) less than or equal to 20 ng/mL. As a result, of low bone density, patients may develop osteonecrosis, chronic inflammation and related pain. This study will be coordinated with patients' regularly scheduled visits for medical care and will require patients to submit blood sample at the start of the study and at 3, 6, 9, AND 12 month visits. Patients will also be scheduled for a bone density measurement (DXA scan) at the start of the study and after 12 months of supplementation to assess for any bone re-mineralization. Thus, the main purpose of this study is to find the amount of nutritional vitamin D that needs to be taken by patients with sickle cell disease in order to correct vitamin D deficiency. The study will also test whether vitamin D supplements improve bone health and reduce inflammation.