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ClinicalTrials.gov public records Last synced Oct 11, 2026, 5:38 AM EDT

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Showing 49–72 of 132 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Sickle Cell Disease & Thalassemia Clear all
Completed No phase listed Observational

Genes Influencing Iron Overload State

NCT01158794
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Marrow Aplasia
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Sep 18, 2019 · Synced Oct 11, 2026, 5:38 AM EDT
Active, not recruiting Phase 1 Interventional

Gene Transfer for Sickle Cell Disease

NCT03282656
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
3 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
2
States / cities
Los Angeles, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 11, 2026, 5:38 AM EDT
Completed No phase listed Observational

Red Blood Cell - IMProving trAnsfusions for Chronically Transfused Recipients

NCT05255445
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Pediatric Cancer
Interventions
Red Blood Cell (RBC) Transfusion
Biological
Lead sponsor
Westat
Other
Eligibility
Not listed
Enrollment
157 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2024
U.S. locations
9
States / cities
Oakland, California • San Francisco, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 17, 2024 · Synced Oct 11, 2026, 5:38 AM EDT
Terminated Phase 1 Interventional Results available

Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases

NCT02231710
View directory record Official record
Conditions
Primary Immune Deficiency Disorders, Hemophagocytic Lymphohistiocytosis, Inherited Bone Marrow Failure Syndrome, Hemoglobinopathies, Metabolic Disorders
Interventions
BPX-501 and Rimiducid
Biological
Lead sponsor
Bellicum Pharmaceuticals
Industry
Eligibility
4 Months to 55 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Mar 25, 2024 · Synced Oct 11, 2026, 5:38 AM EDT
Completed Not applicable Interventional

Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study

NCT04678037
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Hemoglobin C, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia
Interventions
PROs assessment using sickle cell disease mobile app (SCD-app)
Behavioral
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 25 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 21, 2020 · Synced Oct 11, 2026, 5:38 AM EDT
Terminated Phase 2 Interventional Results available

Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease

NCT01000155
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia
Interventions
vorinostat
Drug
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2014
U.S. locations
3
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 21, 2017 · Synced Oct 11, 2026, 5:38 AM EDT
Completed Phase 1 Interventional

Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood Disorders

NCT01316796
View directory record Official record
Conditions
Sickle Cell Anemia, Sickle Cell Disease, Chronic Hemolytic Disorders
Interventions
Sodium Nitrite Cream
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 22, 2018 · Synced Oct 11, 2026, 5:38 AM EDT
Completed Not applicable Interventional

Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App

NCT04675645
View directory record Official record
Conditions
Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
Interventions
HU-Go app
Other
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 25 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 29, 2021 · Synced Oct 11, 2026, 5:38 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

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

NCT03937817
View directory record Official record
Conditions
Alpha and Beta Thalassemia, Sickle Cell Disease, Malaria, Human Physiology
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 70 Years
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 11, 2026, 5:38 AM EDT
Completed No phase listed Observational

Bone Marrow for Hemoglobinopathy Research

NCT00669305
View directory record Official record
Conditions
Sickle Cell Anemia, Thalassemia
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 25, 2020 · Synced Oct 11, 2026, 5:38 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

NCT01931644
View directory record Official record
Conditions
All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions
Not listed
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 100 Years
Enrollment
17,667 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2024
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Apr 18, 2024 · Synced Oct 11, 2026, 5:38 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Allogeneic SCT of NiCord®, UCB-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients With Hemoglobinopathies

NCT01590628
View directory record Official record
Conditions
Sickle Cell Disease & Thalassemia
Interventions
NiCord
Drug
Lead sponsor
Gamida Cell ltd
Industry
Eligibility
2 Years to 45 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2019
U.S. locations
3
States / cities
New York, New York • Durham, North Carolina • Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 20, 2022 · Synced Oct 11, 2026, 5:38 AM EDT
Not listed No phase listed Observational

A Single-arm Safety Study of Transplantation Using Umbilical Cord Blood and Human Placental-derived Stem Cells From Partially Matched Related Donors in Persons With Certain Malignant Blood Diseases and Non-malignant Disorders

NCT00596999
View directory record Official record
Conditions
Hematologic Malignancies
Interventions
UCB and HPDSC
Procedure
Lead sponsor
Celgene Corporation
Industry
Eligibility
Up to 55 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
New Orleans, Louisiana
Source: ClinicalTrials.gov public record
Updated Apr 10, 2012 · Synced Oct 11, 2026, 5:38 AM EDT
Recruiting Phase 1Phase 2 Interventional

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

NCT07599176
View directory record Official record
Conditions
Sickle Cell Disease, Beta-thalassemia
Interventions
Total Body Irradiation, Alemtuzumab, Abatacept, Research blood draw
Radiation · Drug · Other
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 65 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 4, 2026 · Synced Oct 11, 2026, 5:38 AM EDT
Terminated Phase 1 Interventional

Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

NCT07431398
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Pociredir
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years to 65 Years
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
6
States / cities
Miami, Florida • Orlando, Florida • Riverdale, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 18, 2026 · Synced Oct 11, 2026, 5:38 AM EDT
Recruiting Phase 1 Interventional

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

NCT01245179
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
panobinostat
Drug
Lead sponsor
Abdullah Kutlar
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2027
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 11, 2026, 5:38 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

NCT05357482
View directory record Official record
Conditions
Sickle Cell Anemia, Beta Thalassemia
Interventions
TBI, Hydroxyurea, briquilimab, Filgrastim (G-CSF), Sirolimus, Alemtuzumab, Plerixafor
Radiation · Drug · Biological
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 100 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 11, 2026, 5:38 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Haploidentical PBMC Transplant for Severe Congenital Anemias

NCT00977691
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
PBSC Transplant, Alemtuzumab, Sirolimus, Cyclophosphamide, Low Dose Irradiation
Procedure · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Oct 11, 2026, 5:38 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Decision Aid for Therapeutic Options In Sickle Cell Disease

NCT02326597
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia, Hemoglobin SS, Hemoglobin SC, Hemoglobin Beta Thalassemia
Interventions
Decision Aid Tool, Standard Practice
Other
Lead sponsor
Emory University
Other
Eligibility
8 Years to 80 Years
Enrollment
134 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2017
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated Oct 9, 2018 · Synced Oct 11, 2026, 5:38 AM EDT
Completed Not applicable Interventional

The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.

NCT04691323
View directory record Official record
Conditions
Sickle Cell Disease, Sickle B+ Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Hemoglobin C
Interventions
HU-Go app
Other
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 21 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 11, 2026, 5:38 AM EDT
Completed Phase 2 Interventional Results available

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

NCT00919503
View directory record Official record
Conditions
Non-Neoplastic Hematologic and Lymphocytic Disorder
Interventions
Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Methotrexate, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation, Treosulfan, Umbilical Cord Blood Transplantation
Procedure · Biological · Drug + 2 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 49 Years
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
6
States / cities
Aurora, Colorado • Portland, Oregon • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2021 · Synced Oct 11, 2026, 5:38 AM EDT
Completed Not applicable Interventional Results available

Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

NCT04523376
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia Major
Interventions
CliniMACS
Device
Lead sponsor
Timothy Olson
Other
Eligibility
2 Years to 25 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced Oct 11, 2026, 5:38 AM EDT
Completed Phase 3 Interventional Results available

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

NCT00235391
View directory record Official record
Conditions
Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
1,683 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
27
States / cities
Little Rock, Arkansas • Berkeley, California • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jun 7, 2011 · Synced Oct 11, 2026, 5:38 AM EDT
Active, not recruiting No phase listed Observational

Epidemiology of Silent and Overt Strokes in Sickle Cell Disease

NCT03376893
View directory record Official record
Conditions
Anemia, Sickle Cell, Sickle Cell Disease, Stroke, Sickle Cell Thalassemia, Sickle Cell-Beta0-Thalassemia
Interventions
Not listed
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
18 Years and older
Enrollment
102 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2026
U.S. locations
4
States / cities
Birmingham, Alabama • St Louis, Missouri • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 13, 2026 · Synced Oct 11, 2026, 5:38 AM EDT