Sickle Cell Disease Clinical Trials

91 studies are recruiting for sickle cell disease in the United States right now. Each one is listed on ClinicalTrials.gov, the public registry the research teams themselves file with. TrialBoost is not a doctor, a research site, or a study sponsor: the research team decides who takes part.

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Studies now recruiting

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

   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 …

   Stage

   Phase 1

   Ages

   18 to 65

   Site

   National Institutes of Health Clinical Center, Bethesda, Maryland

   Run by

   National Heart, Lung, and Blood Institute (NHLBI)

   See if you can join NCT05904093 on ClinicalTrials.gov

2. A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)

   The primary objective of this study is to determine the effect of mitapivat versus placebo on the need for transfusions in subjects with SCD.

   Stage

   Phase 3

   Ages

   12 and older

   Site

   Indiana Hemophilia & Thrombosis Center, Indianapolis, Indiana

   Locations

   5 in total

   Run by

   Agios Pharmaceuticals, Inc.

   See if you can join NCT07656415 on ClinicalTrials.gov

3. A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

   The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and preliminary food effect of ITU512 as well as the fetal hemoglobin (HbF)-inducing capacity of ITU512. This will be the first evaluation of the potential therapeutic effect of ITU512 in healthy participants …

   Stage

   Phase 1

   Ages

   12 to 55

   Site

   University of Alabama Birmingham, Birmingham, Alabama

   Locations

   14 in total

   Run by

   Novartis Pharmaceuticals

   See if you can join NCT06546670 on ClinicalTrials.gov

4. Blood Sampling for Research Related to Sickle Cell Disease

   This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throu…

   Stage

   Not phased

   Ages

   18 to 100

   Site

   National Institutes of Health Clinical Center, Bethesda, Maryland

   Run by

   National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

   See if you can join NCT00542230 on ClinicalTrials.gov

5. AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

   Children, adolescents, and young adults with malignant and non-malignant conditionsundergoing an allogeneic stem cell transplantation (AlloSCT) will have the stem cells selected utilizing α/β CD3+/CD19+ cell depletion. All other treatment is standard of care.

   Stage

   Phase 2

   Ages

   0 to 30

   Site

   New York Medical College, Valhalla, New York

   Run by

   Mitchell Cairo

   See if you can join NCT04099966 on ClinicalTrials.gov

6. Trans-auricular Nerve Stimulation as an Innovative Approach to the Treatment of Pain in Pediatric Patients Suffering From Sickle Cell Disease

   The goal of this study is to evaluate the effects of transauricular vagus and trigeminal nerve stimulation on the treatment of pain in children with Sickle Cell Disease. * The primary objective is to determine the feasibility of using a tAN device to treat pain in pediatric participants with SCD by…

   Stage

   Not phased

   Ages

   7 to 18

   Site

   Children's Hospital of Atlanta: Eagelston, Atlanta, Georgia

   Run by

   Emory University

   See if you can join NCT07090668 on ClinicalTrials.gov

7. Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease

   A promising approach for the treatment of genetic diseases is called gene therapy. Gene therapy is a relatively new field of medicine that uses genetic material (mostly deoxyribonucleic acid (DNA)) from the patient to treat his or her own disease. In gene therapy, the investigators introduce new ge…

   Stage

   Phase 1

   Ages

   13 to 35

   Site

   Boston Children's Hospital, Boston, Massachusetts

   Run by

   Daniel Bauer

   See if you can join NCT07708350 on ClinicalTrials.gov

8. Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

   This is an open label, non-randomized, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will …

   Stage

   Phase 1

   Ages

   12 to 35

   Site

   University of California, Davis, Davis, California

   Locations

   3 in total

   Run by

   Mark Walters, MD

   See if you can join NCT04774536 on ClinicalTrials.gov

9. Virtual Reality for SCD VOC

   This randomized pilot study will evaluate the feasibility, tolerability, and preliminary analgesic effect of headset-based virtual reality (VR) interventions for adults with sickle cell disease (SCD) experiencing vaso-occlusive crisis (VOC) treated in an infusion center. Up to 50 participants will …

   Stage

   Not phased

   Ages

   18 to 88

   Site

   University of Maryland Medical Center, Stoler Infusion Center, Baltimore, Maryland

   Run by

   University of Maryland, Baltimore

   See if you can join NCT07674277 on ClinicalTrials.gov

10. A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE)

    A phase III, multi-center, randomized, placebo-controlled, double-blind study to assess efficacy and safety of crizanlizumab (5 mg/kg) versus placebo, with or without hydroxyurea/hydroxycarbamide therapy, in adolescent and adult Sickle Cell Disease patients with frequent vaso-occlusive crises.

    Stage

    Phase 3

    Ages

    12 to 100

    Site

    University Of Alabama, Birmingham, Alabama

    Locations

    36 in total

    Run by

    Novartis Pharmaceuticals

    See if you can join NCT06439082 on ClinicalTrials.gov

11. Gene Editing For 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 sympt…

    Stage

    Phase 1

    Ages

    18 to 24

    Site

    St. Jude Children's Research Hospital, Memphis, Tennessee

    Run by

    St. Jude Children's Research Hospital

    See if you can join NCT06506461 on ClinicalTrials.gov

12. The Efficacy and Safety of Rilzabrutinib in Participants Aged 10 to 65 Years With 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 detai…

    Stage

    Phase 3

    Ages

    10 to 65

    Site

    University of Alabama at Birmingham- Site Number : 8400003, Birmingham, Alabama

    Locations

    63 in total

    Run by

    Sanofi

    See if you can join NCT06975865 on ClinicalTrials.gov

13. Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

    This is a non-ablative (partial) stem cell transplant for patients with severe sickle cell disease or beta-thalassemia requiring red cell transfusions. The intensity of the transplant is slightly increased from our previous transplant regimens. The goal is to aim for higher percentage of donor cell…

    Stage

    Phase 1

    Ages

    4 to 65

    Site

    National Institutes of Health Clinical Center, Bethesda, Maryland

    Run by

    National Heart, Lung, and Blood Institute (NHLBI)

    See if you can join NCT07599176 on ClinicalTrials.gov

14. Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain

    The study, known as the Peer suppoRt for adolescents and Emerging adults with Sickle cell pain: promoting ENgagement in Cognitive behavioral thErapy (PRESENCE), aims to determine the effectiveness of digital CBT in reducing pain, opioid use, and healthcare utilization among AYAs with SCD. It also s…

    Stage

    Not phased

    Ages

    16 to 39

    Site

    University of South Alabama Medical Center, Mobile, Alabama

    Locations

    14 in total

    Run by

    University of Pittsburgh

    See if you can join NCT06374238 on ClinicalTrials.gov

15. A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD)

    This is an open-label, multicenter, within-participant dose-escalation study examining up to 3 dose levels of DISC-3405 and will assess the safety, tolerability, PK, and PD of DISC 3405 in participants with sickle cell disease.

    Stage

    Phase 1

    Ages

    18 and older

    Site

    University of Alabama at Birmingham, Birmingham, Alabama

    Locations

    11 in total

    Run by

    Disc Medicine, Inc

    See if you can join NCT07187973 on ClinicalTrials.gov

16. Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

    Background: Blood disorders like sickle cell disease and malaria affect many people around the world. Researchers want to learn more about blood disorders. To do this, they need to collect biological samples from people with blood disorders. They also need to collect samples from healthy people. Ob…

    Stage

    Not phased

    Ages

    18 to 70

    Site

    National Institutes of Health Clinical Center, Bethesda, Maryland

    Run by

    National Institute of Allergy and Infectious Diseases (NIAID)

    See if you can join NCT03937817 on ClinicalTrials.gov

17. Exercise in Child Health

    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 affi…

    Stage

    Not phased

    Ages

    10 to 17

    Site

    University of California, Irvine, Irvine, California

    Run by

    University of California, Irvine

    See if you can join NCT05359991 on ClinicalTrials.gov

18. A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease

    A randomized control trial in 20 subjects with sickle cell disease comparing oral THU-decitabine to nicotinamide and in combination (THU, decitabine and nicotinamide).

    Stage

    Phase 1

    Ages

    18 and older

    Site

    University of Illinois at Chicago College of Medicine, Chicago, Illinois

    Run by

    EpiDestiny, Inc.

    See if you can join NCT04055818 on ClinicalTrials.gov

19. Determination of Red Cell Survival in Sickle Cell Disease and Other Hemoglobinopathies Using Biotin Labeling

    Background: Sickle cell disease (SCD) is an inherited disorder of the blood. SCD causes red blood cells (RBCs) to die early. This can lead to a shortage of healthy cells. SCD and other blood disorders can be managed with drugs or cured with a bone marrow transplant. Researchers want to know how lon…

    Stage

    EARLY_Phase 1

    Ages

    18 to 100

    Site

    National Institutes of Health Clinical Center, Bethesda, Maryland

    Run by

    National Heart, Lung, and Blood Institute (NHLBI)

    See if you can join NCT06313398 on ClinicalTrials.gov

20. Dietary Intake and Dietary Behaviors in Adults With Sickle Cell Disease

    Background: Sickle Cell Disease (SCD) causes blood cells form a crescent shape. It is caused by a genetic mutation in the hemoglobin gene. People with SCD are at increased risk for illnesses like stroke, chronic pain, and heart problems, as well as decreased overall health and well-being. Researche…

    Stage

    Not phased

    Ages

    18 to 120

    Site

    National Institutes of Health Clinical Center, Bethesda, Maryland

    Run by

    National Institutes of Health Clinical Center (CC)

    See if you can join NCT05170412 on ClinicalTrials.gov

21. A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

    Etavopivat is a new medicine under development for treating blood disorders like sickle cell disease and thalassaemia. Sickle cell disease and thalassaemia are inherited blood disorders that affect haemoglobin. Haemoglobin is the protein that carries oxygen through the body. This study is looking i…

    Stage

    Phase 3

    Ages

    2 and older

    Site

    Children's Hospital Los Angeles - Endocrinology, Los Angeles, California

    Locations

    107 in total

    Run by

    Novo Nordisk A/S

    See if you can join NCT06609226 on ClinicalTrials.gov

22. A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease

    This study is conducted to confirm whether etavopivat works well at reducing the number of Vaso-occlusive crisis VOCs (sickle cell pain crises) caused by obstructions in blood vessels in adults and adolescents living with sickle cell disease. The study will also evaluate how well etavopivat can red…

    Stage

    Phase 3

    Ages

    12 and older

    Site

    Uni of Alabama at Birmingham, Birmingham, Alabama

    Locations

    175 in total

    Run by

    Novo Nordisk A/S

    See if you can join NCT06612268 on ClinicalTrials.gov

23. Tocilizumab for Acute Chest Syndrome

    The investigators are evaluating the role of a low dose of tocilizumab in treating acute chest syndrome in patients with sickle cell disease. Tocilizumab inhibits interleukin-6 (IL-6) receptors and is used to treat rheumatoid arthritis and severe cytokine release syndrome, which can be seen with ch…

    Stage

    Phase 2

    Ages

    12 and older

    Site

    University of Chicago, Chicago, Illinois

    Run by

    University of Chicago

    See if you can join NCT05640271 on ClinicalTrials.gov

24. Sickle Cell Disease Biofluid Chip Technology (SCD BioChip)

    '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, sh…

    Stage

    Not phased

    Ages

    12 and older

    Site

    University Hospitals Case Medical Center, Cleveland, Ohio

    Run by

    University Hospitals Cleveland Medical Center

    See if you can join NCT02824471 on ClinicalTrials.gov

25. Study to Understand the Genetic Risk of Developing an Immune Response After Blood Transfusions Among Individuals With 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 …

    Stage

    Not phased

    Ages

    2 to 99

    Site

    National Institutes of Health Clinical Center, Bethesda, Maryland

    Run by

    National Human Genome Research Institute (NHGRI)

    See if you can join NCT06944067 on ClinicalTrials.gov