Sickle Cell Disease Clinical Trials

92 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, page 2

1. 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

   3 in total

   Run by

   Agios Pharmaceuticals, Inc.

   See if you can join NCT07656415 on ClinicalTrials.gov

2. Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease

   The purpose of this study is to determine whether giving extra arginine to patients with sickle cell disease seeking treatment for vaso-occlusive painful events (VOE) will decrease pain scores, decrease need for pain medications or decrease length of hospital stay or emergency department visit.

   Stage

   Phase 1

   Ages

   7 to 21

   Site

   Children's Healthcare fo Atlanta at Hughes Spalding, Atlanta, Georgia

   Locations

   2 in total

   Run by

   Emory University

   See if you can join NCT02447874 on ClinicalTrials.gov

3. 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

   53 in total

   Run by

   Sanofi

   See if you can join NCT06975865 on ClinicalTrials.gov

4. Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

   This is a follow-up trial to NYMC 526 (NCT01461837) to assess the safety, efficacy and toxicity of administering Defibrotide prophylaxis for high-risk sickle cell or beta thalassemia patients undergoing a familial haploidentical or MUD allogeneic stem cell transplantation with CD34 enrichment and T…

   Stage

   Phase 2

   Ages

   0 to 21

   Site

   University of California Los Angeles, Los Angeles, California

   Locations

   4 in total

   Run by

   New York Medical College

   See if you can join NCT02675959 on ClinicalTrials.gov

5. Cooperative Assessment of Late Effects for SCD Curative Therapies

   Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States. Mortality for children with SCD has decreased substantially over the past 4 decades, with >…

   Stage

   Not phased

   Ages

   4 to 65

   Site

   Vanderbilt University Medical Center, Nashville, Tennessee

   Locations

   5 in total

   Run by

   Vanderbilt University Medical Center

   See if you can join NCT05153967 on ClinicalTrials.gov

6. Haploidentical Donor Hematopoietic Cell Transplant for 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 great…

   Stage

   Phase 2

   Ages

   Up to 22

   Site

   St Jude Children's Research Hospital, Memphis, Tennessee

   Run by

   St. Jude Children's Research Hospital

   See if you can join NCT07616154 on ClinicalTrials.gov

7. Alendronate for Osteonecrosis in Adults With Sickle Cell Disease

   A prospective, single-arm, intervention study of oral alendronate in adults with sickle cell disease and osteonecrosis

   Stage

   Phase 2

   Ages

   18 to 80

   Site

   UC Davis Comprehensive Cancer Center, Sacramento, California

   Run by

   University of California, Davis

   See if you can join NCT06016634 on ClinicalTrials.gov

8. Early Life Exposures Among Children With Sickle Cell Disease

   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 resil…

   Stage

   Not phased

   Ages

   18 to 50

   Site

   University of Alabama at Birmingham, Birmingham, Alabama

   Run by

   University of Alabama at Birmingham

   See if you can join NCT05377372 on ClinicalTrials.gov

9. Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

   The study is a Phase II clinical trial. Patients will receive intensity modulated total body irradiation (TBI) at a dose of 3 Gy with standard fludarabine/ i.v. cyclophosphamide conditioning prior to human leukocyte antigen (HLA)-haploidentical hematopoietic stem cell transplant (HSCT). The primary…

   Stage

   Phase 2

   Ages

   16 to 60

   Site

   University of Illinois at Chicago, Chicago, Illinois

   Run by

   University of Illinois at Chicago

   See if you can join NCT03121001 on ClinicalTrials.gov

10. Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe 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 pharmacodynam…

    Stage

    Phase 1

    Ages

    12 to 50

    Site

    Children's Hospital Los Angeles, Los Angeles, California

    Locations

    6 in total

    Run by

    Kamau Therapeutics

    See if you can join NCT04819841 on ClinicalTrials.gov

11. Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease

    This study will examine sleep disordered breathing and sleep quality in participants (ages 12-25) diagnosed with sickle cell disease of any genotype. We will utilize remote peripheral arterial tonometry (PAT) and questionnaires to evaluate difficulties with sleep. PAT assessments will occur remotel…

    Stage

    Not phased

    Ages

    12 to 25

    Site

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

    Run by

    St. Jude Children's Research Hospital

    See if you can join NCT06477289 on ClinicalTrials.gov

12. Hydroxyurea Exposure Limiting Pregnancy and Follow-Up Lactation

    The purpose of this research study is to document and understand the effects of hydroxyurea exposure for women with SCD and their babies, during both gestation and lactation.

    Stage

    Not phased

    Ages

    Any age

    Site

    Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio

    Run by

    Children's Hospital Medical Center, Cincinnati

    See if you can join NCT04093986 on ClinicalTrials.gov

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

    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). 60 AYAs with SCD ages 15-25 and their caregivers and 8 SCD providers will participate in the pilot pragm…

    Stage

    Not phased

    Ages

    15 and older

    Site

    Nemours Children's Hospital, Delaware, Wilmington, Delaware

    Run by

    Nemours Children's Clinic

    See if you can join NCT05383911 on ClinicalTrials.gov

14. Investigation of the Genetics of Hematologic Diseases

    The purpose of this study is to collect and store samples and health information for current and future research to learn more about the causes and treatment of blood diseases. This is not a therapeutic or diagnostic protocol for clinical purposes. Blood, bone marrow, hair follicles, nail clippings…

    Stage

    Not phased

    Ages

    Any age

    Site

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

    Run by

    St. Jude Children's Research Hospital

    See if you can join NCT02720679 on ClinicalTrials.gov

15. Rollover Study for Patients With Sickle Cell Disease Who Have Completed a Prior Novartis-Sponsored Crizanlizumab Study

    This is a multi-center multi-national rollover study to allow continued access to crizanlizumab for patients with sickle cell disease (SCD) who are on crizanlizumab treatment in a Novartis-sponsored study (parent study) and are benefiting from the treatment as judged by the investigator.

    Stage

    Phase 4

    Ages

    0 to 100

    Site

    University Of Alabama, Birmingham, Alabama

    Locations

    31 in total

    Run by

    Novartis Pharmaceuticals

    See if you can join NCT04657822 on ClinicalTrials.gov

16. Sickle Cell Clinical Research and Intervention Program

    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 i…

    Stage

    Not phased

    Ages

    Any age

    Site

    Children's Hospital of Illinois at OSF-Saint Francis Medical Center, Peoria, Illinois

    Locations

    6 in total

    Run by

    St. Jude Children's Research Hospital

    See if you can join NCT02098863 on ClinicalTrials.gov

17. Let's Get REAL: Family Health Communication Tool in Pediatric Stem Cell Transplant and Cellular Therapy

    The investigators will conduct a pilot feasibility and efficacy trial of a newly developed family health communication tool (called Let's Get REAL) in increasing youth involvement in real-time stem cell transplant and cellular therapy decisions (SCTCT). The investigators will pilot the intervention…

    Stage

    Not phased

    Ages

    8 and older

    Site

    Washington University School of Medicine, St Louis, Missouri

    Run by

    Washington University School of Medicine

    See if you can join NCT06689800 on ClinicalTrials.gov

18. Allo HSCT for High Risk Hemoglobinopathies

    A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.

    Stage

    Phase 2

    Ages

    Up to 55

    Site

    Masonic Cancer Center, Minneapolis, Minnesota

    Run by

    Masonic Cancer Center, University of Minnesota

    See if you can join NCT06872333 on ClinicalTrials.gov

19. Ruxolitinib-Enhanced Haplo HCT for Children and Young Adults With Sickle Cell Disease

    This trial will determine whether adding ruxolitinib to a reduced intensity conditioning (RIC) regimen reduces the rate of graft failure following haploidentical (haplo) hematopoietic cell transplant (HCT) for children and young adults with sickle cell disease (SCD). This study will enroll and trea…

    Stage

    Phase 1

    Ages

    12 to 45

    Site

    Children's Healthcare of Atlanta, Atlanta, Georgia

    Locations

    4 in total

    Run by

    Arkansas Children's Hospital Research Institute

    See if you can join NCT07252050 on ClinicalTrials.gov

20. Functional Ovarian Reserve in 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 …

    Stage

    Not phased

    Ages

    10 to 18

    Site

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

    Run by

    St. Jude Children's Research Hospital

    See if you can join NCT07392216 on ClinicalTrials.gov

21. A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

    Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activit…

    Stage

    Phase 2

    Ages

    2 to 50

    Site

    Memorial Sloan Kettering at Basking Ridge (Consent only), Basking Ridge, New Jersey

    Locations

    6 in total

    Run by

    Memorial Sloan Kettering Cancer Center

    See if you can join NCT05736419 on ClinicalTrials.gov

22. Effect of Virtual Reality Technology for Pain Management of Vaso-Occlusive Crisis in Patients With Sickle Cell Disease

    Acute vaso-occlusive crisis (VOC) is the most common complication in patients with sickle cell disease (SCD) and pain related to VOC is often inadequately treated. This is a phase II randomized controlled clinical trial evaluating the efficacy of virtual reality technology when added to standard pa…

    Stage

    Not phased

    Ages

    6 to 25

    Site

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

    Locations

    2 in total

    Run by

    St. Jude Children's Research Hospital

    See if you can join NCT03353584 on ClinicalTrials.gov

23. Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With 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…

    Stage

    Phase 1

    Ages

    1 to 30

    Site

    City of Hope Medical Center, Duarte, California

    Run by

    City of Hope Medical Center

    See if you can join NCT03279094 on ClinicalTrials.gov

24. SickleFit Exercise and Nutrition Study

    To goal of this study is to pilot the SickleFit exercise and nutrition intervention in adults with sickle cell disease in a randomized control trial

    Stage

    Not phased

    Ages

    35 and older

    Site

    Duke University, Durham, North Carolina

    Run by

    Duke University

    See if you can join NCT07442851 on ClinicalTrials.gov

25. Relationship Between Abnormal Myocardial Perfusion and Diastolic Dysfunction in Sickle Cell Disease Using PET

    There is limited information on what causes injury to the heart in individuals with Sickle Cell Disease (SCD). Researchers in this study want to see if decreased blood flow to the heart during stress could be causing the heart damage seen in SCD patients. They also want to test people who don't hav…

    Stage

    Phase 2

    Ages

    18 to 21

    Site

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

    Run by

    St. Jude Children's Research Hospital

    See if you can join NCT05583721 on ClinicalTrials.gov