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Sickle Cell Disease clinical trials near you

A clinical trial is a research study that tests whether a treatment is safe and works. Taking part is always your choice, and you can stop at any time. Listing a study here is not a recommendation — talk it through with your own team.

138 recruiting studiesFrom ClinicalTrials.gov (opens in a new tab)

Trial results

8 shown of 138

8 shown of 138 clinical trials.

  1. RecruitingPhase 1InterventionalNCT06442761

    SCD Stem Cell Mobilization and Apheresis Using Motixafortide

    Sickle Cell Disease

    This study is being done to see if the study drug, motixafortide, is safe in participants with sickle cell disease (SCD). Investigators also want to see if the drug will help the body increase the number of stem cells that can be collected for possible future transplant use. PRIMARY OBJECTIVE * To characterize the safety and tolerability of motixafortide in participants with SCD as determined by the incidence of adverse events (AEs). SECONDARY OBJECTIVES * To characterize the efficacy of a single dose (Part A) or two doses (Part B) of motixafortide for hematopoietic stem cell (HSC) mobilization and apheresis collection in participants with SCD as determined by the yield of CD34+ cells (CD34+ cells/kg). * To measure the mobilization effects of single-day (Part A) or daily dosing (Part B) dosing with motixafortide in the peripheral blood in participants with SCD as determined by peak peripheral blood CD34+ counts * To recommend a phase 2 dosing strategy based on safety, efficacy, and mobilization effects

    Sites
    3
    Target
    20
    Study sites
    Birmingham, US +2 more

    Sponsor: St. Jude Children's Research Hospital

  2. RecruitingInterventionalNCT06367192

    School Readiness Intervention for Preschool Children With Sickle Cell Disease

    Sickle Cell Disease

    The study participant is being asked to take part in this clinical trial, a type of research study, because the participant is a young child with sickle cell disease or the caregiver of a child with sickle cell disease. This study is being done to test a school readiness program for children with sickle cell disease (ages 3.5-6,5 years old). Primary Objective Assess feasibility and acceptability of an adapted school readiness intervention among preschool children (ages 3.5-6.5) diagnosed with sickle cell disease. Secondary Objectives Objective 1: Measure preliminary efficacy of the adapted school readiness intervention compared to routine care among preschool children ages (3.5-6.5) diagnosed with sickle cell disease. Objective 2: Examine implementation factors (i.e., barriers and facilitators) during post-intervention.

    Sites
    1
    Target
    36
    Study sites
    Memphis, US

    Sponsor: St. Jude Children's Research Hospital

  3. RecruitingObservationalNCT07116772

    Serial Assessment of Fertility Experiences

    Sickle Cell Anemia

    The SAFE study is a long-term research project that watches people with sickle cell anemia (SCA) over time. The main goal is to see how a medicine called hydroxyurea affects their growth, puberty, and ability to have children. A second goal is to see how hydroxyurea affects pregnancy outcomes, by comparing people who take the medicine to those who don't.

    Sites
    3
    Target
    250
    Study sites
    Santo Domingo, DO +2 more

    Sponsor: Children's Hospital Medical Center, Cincinnati

  4. RecruitingObservationalNCT02098863

    Sickle Cell Clinical Research and Intervention Program

    Sickle Cell Disease

    Despite the important work of previous sickle cell disease (SCD) cohort studies, there remain many understudied areas that require investigation. An important knowledge deficit is the slow but progressive process of chronic end-organ dysfunction. The majority of organ dysfunction becomes apparent in the young adult years, but comprehensive assessment of adults and understanding of predictors of adulthood organ dysfunction are insufficient. Similarly, the role of disease-modifying therapies, such as hydroxyurea, in preventing organ dysfunction later in life is not clear. Extended follow-up of patients through the transition into adulthood is imperative to understand the long-term implications of pediatric sickle cell care. This observational study will collect data in a systematic fashion at participants' regular clinic visits (in-person or remote) to answer the objectives described below. In addition to primary study objectives, SCCRIP participants will be eligible to participate in a sub-study, which will investigate genetically determined responses to Hydroxyurea (HU) via a pharmacokinetic study (PK). This one time study will involve blood collection at timed intervals proceeding a dose of HU. Defining the basis for this inter-individual variability will allow the identification of poor HU responders prior to initiation of therapy and the seeking of alternative treatments which seek to optimize disease treatment by accounting for individual variability in genes, environment, and lifestyle.

    Sites
    6
    Target
    10,000
    Study sites
    Peoria, US +5 more

    Sponsor: St. Jude Children's Research Hospital

  5. RecruitingObservationalNCT04416178

    Sickle Cell Disease and the Genomic and Gene Therapy Needs of Stakeholders

    Sickle Cell Disease

    The primary objectives of this prospective mixed-method interview study are to use semi-structured interviews in parents of sickle cell disease (SCD) patients to describe parental attitudes of research involving genomic sequencing, including concerns about participation and expectations from researchers and second, to use surveys to quantitatively measure genetic/genomic knowledge, trust in health care provider, and literacy/numeracy ability in parents of children with SCD and adolescents with SCD. Secondary objectives are development of a web-based tool about treatment options for SCD that fosters patient-clinician communication and promotes shared decision-making. The web-based tool will undergo usability and pilot testing to ensure it is accessible to families and provide data about strategies for integrating into clinical conversations about treatment options. Investigators will use the data generated to reduce the risk of misunderstanding about DNA and genetic research and build strong relationships between SCD families and researchers in the future. The project will design educational information and study materials to help parents of children with SCD understand important details about genomic medicine in SCD care.

    Sites
    1
    Target
    352
    Study sites
    Memphis, US

    Sponsor: St. Jude Children's Research Hospital

  6. RecruitingObservationalNCT02824471

    Sickle Cell Disease Biofluid Chip Technology (SCD BioChip)

    Sickle Cell Disease

    'Sickle-shaped' anemia was first clinically described in the US in 1910, and the mutated heritable sickle hemoglobin molecule was identified in 1949. The pathophysiology of SCD is a consequence of abnormal polymerization of sickle hemoglobin (HbS) and its effects on red cell membrane properties, shape, and density, and subsequent critical changes in inflammatory cell and endothelial cell function. Our goal is to understand the impact of CMA abnormalities in SCD, by interrogating a number of recognized interactions in a range of clinical phenotypes. To date, correlative studies in SCD, by us and others, have range between clinical reports, based on tests, interventions, and chart review of individuals or groups of individuals and, at the other extreme, identification of functional gene polymorphisms based on population studies. The investigators wish to augment these studies through a systematic examination of cellular membrane properties and activation status. Of hematologic disorders, SCD may be unusually susceptible to such an examination.

    Sites
    1
    Target
    100
    Study sites
    Cleveland, US

    Sponsor: University Hospitals Cleveland Medical Center

  7. RecruitingPhase 2InterventionalNCT06358638

    Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell Antibody

    Sickle Cell Disease

    This multicenter prospective study seeks to determine if daratumumab given, prior to HLA-identical sibling donor transplantation using alemtuzumab, low dose total-body irradiation, and sirolimus, can prevent pure red blood cell aplasia with an acceptable safety profile in patients with anti-donor red blood cell antibodies, achieving an event-free survival similar to transplanted patients without such antibodies.

    Sites
    1
    Target
    12
    Study sites
    Washington D.C., US

    Sponsor: Children's National Research Institute

  8. RecruitingInterventionalNCT05373771

    Sickle Cell Improvement: Enhancing Care in the Emergency Department

    Sickle Cell Crisis

    Sickle cell disease (SCD) is an inherited blood disorder affecting approximately 36,000 children in the United States, approximately 90% of whom are Black. The disease is characterized by recurrent, severe pain crises which result in high rates of emergency department visits and hospitalizations, and decreased quality of life. The National Heart, Lung and Blood Institute, as well as the American Society of Hematology, have endorsed pain management guidelines regarding the timeliness of care for children presenting with these acute pain crises. These evidence-based guidelines are infrequently followed, resulting in increased pain and hospitalizations. In additional to other barriers to following the guideline, structural racism has been proposed as a significant contributor and the New England Journal of Medicine recently called for the institution of SCD-specific pain management protocols to combat structural racism and reduce time to opioid administration. The investigators' long-term goal is to improve the care and health outcomes of children with acute painful vaso-occlusive crisis treated in the emergency department. The overall aim of the investigators is to test a care pathway using multifaceted implementation strategies to increase guideline adherent care for children in the emergency department with acute painful vaso-occlusive crisis.

    Sites
    1
    Target
    5,328
    Study sites
    Milwaukee, US

    Sponsor: Medical College of Wisconsin