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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. RecruitingInterventionalNCT05099874

    Feasibility and Efficacy of Attentional-Control Training in Sickle Cell Disease

    Sickle Cell Disease, Attention Deficit, Cognitive Deficit in Attention

    Children with sickle cell disease (SCD) exhibit significantly reduced cognitive functioning (often difficulties with attention) compared to peers and siblings without SCD. EndeavorRx (Akili Interactive Labs: Boston, MA) is an FDA-approved home-based, electronic attentional-control training program designed to treat attention problems in youth. Users access EndeavorRx on a tablet device for 25-30 minutes each day, 5 days per week, for 4 weeks. The program involves training in a game-like environment that repeatedly challenges attentional-control abilities and adapts to user performance, becoming more difficult over time as performance improves. This pilot study is examining the feasibility, acceptability, and preliminary efficacy of EndeavorRx in a sample of 20 children with SCD ages 8-16 who are being treated with chronic blood transfusion therapy.

    Sites
    1
    Target
    20
    Study sites
    Washington D.C., US

    Sponsor: Children's National Research Institute

    View on ClinicalTrials.gov(opens in a new tab)
  2. RecruitingObservationalNCT07392216

    Functional Ovarian Reserve in Sickle Cell Disease

    Sickle Cell Disease

    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.

    Sites
    1
    Target
    440
    Study sites
    Memphis, US

    Sponsor: St. Jude Children's Research Hospital

    View on ClinicalTrials.gov(opens in a new tab)
  3. RecruitingPhase 1, Phase 2InterventionalNCT04819841

    Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

    Sickle Cell Disease

    This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

    Sites
    6
    Target
    15
    Study sites
    Los Angeles, US +5 more

    Sponsor: Kamau Therapeutics

    View on ClinicalTrials.gov(opens in a new tab)
  4. RecruitingPhase 1InterventionalNCT06506461

    Gene Editing For Sickle Cell Disease

    Sickle Cell Disease

    This study is being done to test the safety of a new treatment called gene editing in Sickle Cell Disease (SCD) patients and to see if a single dose of this genetically modified cellular product will increase the amount of a certain hemoglobin called fetal hemoglobin (HbF) and help reduce the symptoms of SCD. Primary Objective * To assess the safety of autologous infusion of clustered regularly interspaced palindromic repeats (CRISPR)/ CRISPR associated protein (Cas9)-edited CD34+ hematopoietic stem and progenitor cells (HSPCs) in patients with severe SCD. Secondary Objective * To assess the efficacy autologous infusion of CRISPR/Cas9 genome-edited CD34+ HSPCs into patients with severe SCD.

    Sites
    1
    Target
    25
    Study sites
    Memphis, US

    Sponsor: St. Jude Children's Research Hospital

    View on ClinicalTrials.gov(opens in a new tab)
  5. RecruitingObservationalNCT05810181

    Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

    Sickle Cell Disease

    This prospective mixed-method interview study aims to qualitatively describe the beliefs, attitudes, and informational needs around gene therapy for rare pediatric diseases among patients and parents of children with a rare disease targeted for treatment using gene therapy techniques. Using learned insights, the team will develop an online platform providing educational content and patient decision aids for patients and their families.

    Sites
    1
    Target
    145
    Study sites
    Memphis, US

    Sponsor: St. Jude Children's Research Hospital

    View on ClinicalTrials.gov(opens in a new tab)
  6. RecruitingObservationalNCT03685721

    Genotype -Phenotype Correlation of PKLR Variants With Pyruvate Kinase, 2,3-Diphosphglycerate and Adenosine Triphosphate Activities in Red Blood Cells of People With Sickle Cell Disease

    Sickle Cell, PKLR Variants, Adenosine Triphosphate Activities

    Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms. The PKLR gene helps create a protein, called pyruvate kinase that is essential in normal functioning of the red blood cell. Differences in the PKLR gene, called genetic variants, may cause some changes in the pyruvate kinase protein and other proteins, that can affect functioning of the red blood cell adding to the effect of SCD. Researchers can study these differences by looking at DNA (the material that determines inherited characteristics). Objective: To study how the PKLR gene affects sickle cell disease. Eligibility: Adults ages 18-80 of African descent. They may have sickle cell disease or not. They must not have had a transfusion recently or have a known deficiency of pyruvate kinase. They cannot be pregnant. Design: Participants will be screened with questions. Participants will have blood drawn by needle in an arm vein. The blood will be genetically tested. Not much is known about how genes affect SCD, so the test results will not be shared with participants or their doctors. ...

    Sites
    1
    Target
    800
    Study sites
    Bethesda, US

    Sponsor: National Heart, Lung, and Blood Institute (NHLBI)

    View on ClinicalTrials.gov(opens in a new tab)
  7. RecruitingPhase 2InterventionalNCT07616154

    Haploidentical Donor Hematopoietic Cell Transplant for Sickle Cell Disease

    Sickle Cell Disease

    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.

    Sites
    1
    Target
    45
    Study sites
    Memphis, US

    Sponsor: St. Jude Children's Research Hospital

    View on ClinicalTrials.gov(opens in a new tab)
  8. RecruitingPhase 1InterventionalNCT03279094

    Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

    Sickle Cell Disease

    This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.

    Sites
    1
    Target
    11
    Study sites
    Duarte, US

    Sponsor: City of Hope Medical Center

    View on ClinicalTrials.gov(opens in a new tab)