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Sickle Cell Disease clinical trials

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NCT ID: NCT05384756 Not yet recruiting - Sickle Cell Disease Clinical Trials

TMLI and Alemtuzumab for Treatment of Sickle Cell Disease

Start date: June 25, 2022
Phase: Phase 1
Study type: Interventional

This phase I trial tests the safety and effectiveness of total marrow and lymphoid irradiation (TMLI) and alemtuzumab as a conditioning regimen in patients with sickle cell disease. Conditioning regimens are treatments used to prepare a patient for stem cell transplantation. A stem cell transplant is a procedure in which a person receives blood stem cells, which make any type of blood cell. A conditioning regimen may include chemotherapy, monoclonal antibody therapy, and radiation to the entire body. It helps make room in the patient's bone marrow for new blood stem cells to grow, and helps prevent the patient's body from rejecting the transplanted cells. Alemtuzumab is a monoclonal antibody that may interfere with the ability of cancer cells to grow and spread. Graft-versus-host disease (GVHD) is a complication that may occur after hematopoietic cell transplantation (HCT) in which donated cells view the recipient's cells as foreign and attack them. Giving TMLI and alemtuzumab may help reduce organ damage that can be caused by radiation and decrease the risk of GVHD.

NCT ID: NCT05383911 Not yet recruiting - Sickle Cell Disease Clinical Trials

Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA)

Start date: August 2023
Phase: N/A
Study type: Interventional

This study will evaluate the acceptability, feasibility, and preliminary efficacy of a shared decision making intervention for adolescents and young adults (AYAs) with sickle cell disease (SCD). 30 AYAs with SCD (ages 15-25 years) and their caregivers and 10 healthcare providers will participate in the single-arm pilot trial. Study procedures for this future clinical trial will be specified once intervention development, usability testing, and implementation planning phases (currently underway) have been completed.

NCT ID: NCT05377372 Recruiting - Sickle Cell Disease Clinical Trials

Early Life Exposures Among Children With Sickle Cell Disease

Start date: October 10, 2019
Phase: N/A
Study type: Interventional

This study is being conducted to determine the relationship between early childhood exposures, such as Adverse Childhood Experiences, Social Determinants of Health and nutrition/breastfeeding, among children with sickle cell disease, and behavioral interventions aimed to reshape psychological resilience and lifestyle factors towards positive health outcomes.

NCT ID: NCT05376046 Recruiting - Sickle Cell Disease Clinical Trials

Study of Erythrocyte Parameters and Hypercoagulability in Sickle Cell Disease (SCD-TGA)

Start date: September 1, 2018
Study type: Observational [Patient Registry]

Sickle cell disease (SCD) is an inherited haemoglobinopathy disorder caused by mutations in HBB gene with amino-acid substitution on β globin chain. The consequence is synthesis of altered haemoglobin S (HbS) which polymerises in red blood cell (RBC) at deoxygenated state. SCD is associated with chronic haemolytic anaemia, vaso-occlusive crisis (VOC) leading to frequent hospitalisation. The aim of the study was to to investigate whether a combination of routine laboratory biomarkers of haemolysis could be used to predict VOC development in confirmed SCD patients.

NCT ID: NCT05371184 Recruiting - Sickle Cell Disease Clinical Trials

Glutamine Role in Preventing Vaso-occlusive Crisis Among SCD Patients

Start date: January 4, 2022
Phase: Phase 4
Study type: Interventional

Prospective phase IV interventional open label randomized controlled trial to assess safety and efficacy of glutamine in preventing vaso-occlusive crisis (VOC) episodes in sickle cell pediatrics and adolescents' patients

NCT ID: NCT05359991 Recruiting - Cystic Fibrosis Clinical Trials

Exercise in Child Health

Project REACH
Start date: May 15, 2022
Phase: N/A
Study type: Interventional

This study is a cooperative investigation funded by the NIH. The project is a collaboration among three major NIH Clinical Translational Science Awardees: 1) UCI (lead site with its affiliate CHOC), 2) Northwestern University (with its affiliate Lurie Children's Hospital), and 3) USC (with its affiliate Children's Hospital of Los Angeles). There is an increasing number of children who, through medical advances, now survive diseases and conditions that were once fatal, but which remain chronic and debilitating. A major challenge to improve both the immediate and long term care and health of such children has been the gap in our understanding of how to assess the biological effects of exercise. Like otherwise healthy children, children with chronic diseases and disabilities want to be physically active. The challenge is to determine what constitutes safe and beneficial level of physical activity when the underlying disease or condition [e.g., cystic fibrosis (CF) or sickle cell disease (SCD)] imposes physiological constraints on exercise that are not present in otherwise healthy children. Current exercise testing protocols were based on studies of athletes and high performing healthy individuals and were designed to test limits of performance at very high-intensity, unphysiological, maximal effort. These approaches are not optimal for children and adolescents with disease and disability. This project (REACH-Revamping Exercise Assessment in Child Health) is designed to address this gap. Cohorts of children will be identified with two major genetic diseases (CF and SCD) and measure exercise responses annually as they progress from early puberty to mid or late puberty over a 3-4year period. In addition, in the light of the pandemic, a group of children will be added who were affected by SARS-CoV-2 and investigate their responses to exercise. SARS-CoV-2 has similar long-term symptoms than CF and SCD have. Novel approaches to assessing physiological responses to exercise using advanced data analytics will be examined in relation to metrics of habitual physical activity, circulating biomarkers of inflammation and growth, leukocyte gene expression, and the impact of the underlying CF, SCD or SARS-CoV-2 condition. The data from this study will help to develop a toolkit of innovative metrics for exercise testing that will be made available to the research and clinical community.

NCT ID: NCT05353647 Not yet recruiting - Sickle Cell Disease Clinical Trials

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

Start date: May 2022
Phase: Phase 2
Study type: Interventional

A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine in which genetic material (mostly DNA) in the patient is changed to treat his or her own disease. In gene therapy, we introduce new genetic material in order to fix or replace the patient's disease gene, with the goal of curing the disease. The procedure is similar to a bone marrow transplant, in that the patient's malfunctioning blood stem cells are reduced or eliminated using chemotherapy, but it is different because instead of using a different person's (donor) blood stem cells for the transplant, the patient's own blood stem cells are given back after the new genetic material has been introduced into those cells. This approach has the advantage of eliminating any risk of graft versus host disease (GVHD), reducing the risk of graft rejection, and may also allow less chemotherapy to be utilized for the conditioning portion of the transplant procedure. To introduce new genetic material into the patient's own blood stem cells we use a modified version of a virus (called a 'vector') that efficiently inserts the "correcting" genetic material into the cells. The vector is a specialized biological medicine that has been formulated for use in human beings. Fetal hemoglobin (HbF) is a healthy, non-sickling kind of hemoglobin. The investigators have discovered a gene that is very important in controlling the amount of HbF. Decreasing the expression of this gene in sickle cell patients could increase the amount of fetal hemoglobin while simultaneously reducing the amount of sickle hemoglobin in their blood, specifically the amount in red blood cells where sickle hemoglobin causes damage to the cell, and therefore potentially cure or significantly improve the condition. The gene we are targeting for change in this study that controls the level of fetal hemoglobin is called BCL11A. In summary, the advantages of a gene therapy approach include: 1) it can be used even if the patient does not have a matched donor available; 2) it may allow a reduction in the amount of chemotherapy required to prepare the patient for the transplant; and 3) it will avoid certain strong medicines often required to prevent and treat GVHD and rejection. Our lab studies with normal mice, mice that have a form of SCD, and with cells from the bone marrow of SCD patients who have donated bone marrow for research purposes show this approach is very effective in reducing the amount of sickle hemoglobin in red cells. Our pilot trial testing this approach in 10 patients with SCD has shown that the treatment has not caused any unexpected safety problems, and that it increases HbF within the red blood cells. Our goal is to continue to test whether this approach is safe, and whether using gene therapy to change the expression of BCL11A will lead to decreased episodes of vaso-occlusive crisis pain in people with SCD.

NCT ID: NCT05348915 Enrolling by invitation - Sickle Cell Disease Clinical Trials

A Study to Evaluate the Long-term Safety of Inclacumab Administered to Participants With Sickle Cell Disease

Start date: March 29, 2022
Phase: Phase 3
Study type: Interventional

This study is an open-label study to evaluate the safety of long-term administration of inclacumab in participants with sickle cell disease (SCD). Participants in this study will have completed a prior study of inclacumab.

NCT ID: NCT05336201 Not yet recruiting - Sickle Cell Disease Clinical Trials

Cognitive Remediation Intervention to Prepare for Transition of Care

Start date: July 1, 2022
Phase: Phase 2
Study type: Interventional

Randomized Controlled Trial (RTC) testing the efficacy of a telehealth adaptation of the Cognitive-Remediation of Executive and Adaptive Deficits in Youth (C-READY) intervention to prepare adolescents with sickle cell disease for transition of care.

NCT ID: NCT05334576 Recruiting - Sickle Cell Disease Clinical Trials

Study of Crizanlizumab for Prevention of Silent Cerebral Infarcts in SCA

Start date: April 11, 2022
Phase: N/A
Study type: Interventional

In this prospective, single-arm, open-label, imaging and treatment study, the investigator will test the hypothesis that crizanlizumab will prevent the progression of silent cerebral infarcts in patients with sickle cell disease. Study participants will undergo brain MRI before initiation of crizanlizumab and at 6 and 30 months after starting crizanlizumab infusions. The crizanlizumab cohort will be compared to a matched, observational cohort of patients not receiving crizanlizumab.