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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 1InterventionalNCT01245179

    Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

    Sickle Cell Disease

    The goal of this clinical research study is to find out about the safety and effects of a drug called panobinostat when given to adults with sickle cell disease. Panobinostat is a pan histone deacetylase (HDAC) inhibitor. HDAC inhibitors have been shown to significantly increase hemoglobin F induction, which is well documented to improve outcomes in sickle cell disease. HDAC inhibitors are also known to potently inhibit cell-specific inflammation, which is a primary contributor to the debilitating effects of sickle cell disease. Given the relevance of these mechanisms of action in SCD, panobinostat may prove to contribute significantly to the management of SCD patients, a population in critical need of further effective treatment options.

    Sites
    1
    Target
    18
    Study sites
    Augusta, US

    Sponsor: Abdullah Kutlar

  2. RecruitingObservationalNCT05799118

    Study of the Role of Genetic Modifiers in Hemoglobinopathies

    Sickle Cell Disease, Thalassemia, Beta, Thalassemia Alpha, Hemoglobinopathies

    This study will investigate the role of genetic modifiers in hemoglobinopathies through a large-scale, multi-ethnic genome-wide association study (GWAS).

    Sites
    26
    Target
    30,000
    Study sites
    Boston, US +25 more

    Sponsor: Cyprus Institute of Neurology and Genetics

  3. RecruitingPhase 1InterventionalNCT05904093

    Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

    Sickle Cell Disorders, Sickle Cell Disease, Hemoglobin S, Hb-SS Disease, Disease Sickle Cell Anemia

    Background: Sickle cell disease (SCD) is a genetic disease that causes the body to produce abnormal ( sickled ) red blood cells. SCD can cause anemia and life-threatening complications in the lungs, heart, kidney, and nerves. People with SCD are also at increased risk of forming blood clots in the veins and lungs, but the standard treatments for these clots can cause increased bleeding in people with SCD. Better treatments are needed. Objective: To test a drug (fostamatinib) in people with SCD. Eligibility: People aged 18 to 65 with SCD. Design: Participants will have 6 clinic visits over 12 weeks. Each visit will be 2 to 3 hours. Participants will be screened. They will have a physical exam with blood tests. They will tell the researchers about the medications they take. Fostamatinib is a tablet taken by mouth. Participants will take the drug at home, twice a day, for up to 6 weeks. Participants will have a clinic visit every 2 weeks while they are taking the drug. At each visit they will have a physical exam with blood tests. They will talk about any side effects the drug may be causing. If they are tolerating the drug well after the first 2 weeks, they may begin taking a higher dose. Participants will have a final visit 4 weeks after they stop taking the drug. They will have a physical exam and blood tests; they will be checked for any side effects of the drug.

    Sites
    1
    Target
    35
    Study sites
    Bethesda, US

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

  4. RecruitingObservationalNCT06944067

    Study to Understand the Genetic Risk of Developing an Immune Response After Blood Transfusions Among Individuals With Sickle Cell Disease

    Sickle Cell Disease

    The purpose of this research study is to look at genes and determine how they interact with each other to find changes that could explain why some people's immune systems may respond to blood transfusions. This response is called an alloimmune response. We strongly believe that when someone has an alloimmune response, it is caused by changes in their genes. We plan to compare changes in the genes of individuals that develop red blood cell alloimmunization after blood transfusions with those that do not develop alloimmunization. This may help us to create more targeted therapeutic interventions, which may improve the health of alloimmune responders.

    Sites
    1
    Target
    50
    Study sites
    Bethesda, US

    Sponsor: National Human Genome Research Institute (NHGRI)

  5. RecruitingPhase 2InterventionalNCT07369024

    Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults

    Sickle Cell Disease, Vaso-Occlusive Pain Episode in Sickle Cell Disease, Ketamine Infusion, Pain Management, Vaso-Occlusive Crises

    The purpose of this research is to see if ketamine is effective and safe in treating children and young adults with sickle cell disease experiencing sickle cell related pain. In this study, we will compare the outcomes (such as pain scores) in persons who receive standard of care pain medicine (an opioid such as morphine) plus a low dose (amount) of ketamine to those who receive only standard of care pain medicine.

    Sites
    1
    Target
    120
    Study sites
    Torrance, US

    Sponsor: Mohsen Saidinejad

  6. RecruitingPhase 1, Phase 2InterventionalNCT03653338

    T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

    Sickle Cell Anemia, Beta-Thalassemia Major, Diamond-Blackfan Anemia

    The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting.

    Sites
    1
    Target
    5
    Study sites
    Pittsburgh, US

    Sponsor: Paul Szabolcs

  7. RecruitingPhase 2InterventionalNCT07175051

    Targeting the Pathophysiology of Sickle Cell-Related Kidney Disease Using the SGLT2 Inhibitors, Empagliflozin

    Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Albuminuria

    Sickle cell anemia (SCA) is an inherited red blood disorder. The kidneys are among the most commonly affected organ systems in SCA. The Food and Drug Administration (FDA) has approved empagliflozin as a treatment to reduce the decline of kidney function in those with kidney disease. The proposed research study aims to determine whether empagliflozin can prevent the progression of kidney dysfunction in patients with sickle cell anemia (SCA) who are at high risk of developing advanced chronic kidney disease (CKD).

    Sites
    1
    Target
    20
    Study sites
    Chicago, US

    Sponsor: University of Illinois at Chicago

  8. RecruitingPhase 3InterventionalNCT06975865

    The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With Sickle-cell Disease

    Sickle Cell Disease

    This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, flexible-adaptive, group-sequential study (Part A), followed by an open-label LTE period (Part B) to investigate the efficacy, and safety of rilzabrutinib in participants with sickle-cell disease (SCD). Study details include: * Study duration: a 52-week double-blind period (Part A), followed by an open-label LTE period (Part B). Double-blind period has two parts, 50% (adult only) until the interim analysis (a proof-concept part analogous to a phase 2b study), and 50% (adult and children) after the interim analysis. Only the participants who complete double-blind treatment period (Part A) are eligible to continue to the LTE period. The duration of the LTE period (Part B) will be from the first-participant-in (FPI)-LTE (Part B) until the last participant who enters the LTE has completed 52 weeks. * Treatment duration: 52-week double-blind period (Part A); LTE period (Part B) from the (FPI until the last participant who enters the LTE has completed 52 weeks. * Visit frequency: Week visits based on the Schedule of Assessments.

    Sites
    63
    Target
    192
    Study sites
    Birmingham, US +62 more

    Sponsor: Sanofi