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ClinicalTrials.gov public records Last synced Oct 10, 2026, 3:02 AM EDT

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Showing 25–48 of 132 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Sickle Cell Disease & Thalassemia Clear all
Completed Phase 1 Interventional

Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload

NCT01039636
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-thalassemia
Interventions
FBS0701
Drug
Lead sponsor
FerroKin BioSciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
3
States / cities
Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 10, 2012 · Synced Oct 10, 2026, 3:02 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

The BENeFiTS Trial in Beta Thalassemia Intermedia

NCT04432623
View directory record Official record
Conditions
Beta Thalassemia Intermedia, Sickle Cell Disease
Interventions
Benserazide Only Product
Drug
Lead sponsor
Phoenicia BioScience
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
4
States / cities
Oakland, California • Boston, Massachusetts • Weston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 15, 2025 · Synced Oct 10, 2026, 3:02 AM EDT
Completed No phase listed Observational

Screening Patients With Sickle Cell Disease for Kidney Damage

NCT02239016
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
320 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
4
States / cities
Atlanta, Georgia • Louisville, Kentucky • Bethesda, Maryland + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 29, 2021 · Synced Oct 10, 2026, 3:02 AM EDT
Recruiting No phase listed Observational

ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders

NCT04398628
View directory record Official record
Conditions
Hematologic Disorder, Bleeding Disorder, Connective Tissue Disorder, Hemophilia, Thrombosis, Von Willebrand Diseases, Thrombophilia, Rare Bleeding Disorder, Platelet Disorder, Factor IX Deficiency, Factor VIII Deficiency, Thalassemia, Sickle Cell Disease
Interventions
Not listed
Lead sponsor
American Thrombosis and Hemostasis Network
Network
Eligibility
Not listed
Enrollment
3,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
76
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 60 more
Source: ClinicalTrials.gov public record
Updated Aug 17, 2026 · Synced Oct 10, 2026, 3:02 AM EDT
Completed Phase 4 Interventional

A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

NCT03176849
View directory record Official record
Conditions
Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder, Pediatric Acute Myeloid Leukemia, Pediatric Acute Lymphoid Leukemia, Myelodysplastic Syndromes, Sickle Cell Anemia in Children, Aplastic Anemia, Thalassemia in Children
Interventions
Vitamin D3, Standard Vitamin D3 Supplementation
Dietary Supplement
Lead sponsor
Phoenix Children's Hospital
Other
Eligibility
1 Year to 25 Years
Enrollment
49 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2019
U.S. locations
1
States / cities
Phoenix, Arizona
Source: ClinicalTrials.gov public record
Updated Nov 17, 2020 · Synced Oct 10, 2026, 3:02 AM EDT
Recruiting Early Phase 1 Interventional

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

NCT06313398
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Hemoglobinopathy
Interventions
Biotin-labeled red blood cells
Biological
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 10, 2026, 3:02 AM EDT
Recruiting Phase 2 Interventional

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)

NCT05736419
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Beta, Thalassemia
Interventions
Fludarabine, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Rabbit ATG, Dexamethasone, Bortezomib, Rituximab
Drug · Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
2 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
6
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 3, 2026 · Synced Oct 10, 2026, 3:02 AM EDT
Active, not recruiting Phase 2 Interventional

An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia

NCT04053803
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
IMR-687
Drug
Lead sponsor
Imara, Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
Farmington, Connecticut • Hollywood, Florida • Temple, Texas
Source: ClinicalTrials.gov public record
Updated Mar 4, 2022 · Synced Oct 10, 2026, 3:02 AM EDT
Recruiting No phase listed Observational

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

NCT04528355
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions
Interventions
data collection
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 60 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 10, 2026, 3:02 AM EDT
Terminated Phase 2 Interventional Results available

L-Glutamine Therapy for Sickle Cell Anemia

NCT00586209
View directory record Official record
Conditions
Sickle Cell Anemia, Thalassemia
Interventions
L-Glutamine, Placebo
Drug
Lead sponsor
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Other
Eligibility
18 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
1
States / cities
Torrance, California
Source: ClinicalTrials.gov public record
Updated Feb 2, 2021 · Synced Oct 10, 2026, 3:02 AM EDT
Completed Phase 2 Interventional Accepts healthy volunteers Results available

Glutamine Therapy for Hemolysis-Associated Pulmonary Hypertension

NCT01048905
View directory record Official record
Conditions
Pulmonary Hypertension, Sickle Cell Disease, Thalassemia
Interventions
L-Glutamine
Drug
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
4 Years and older
Enrollment
13 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2014
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Oct 10, 2026, 3:02 AM EDT
Completed Not applicable Interventional Results available

Massive Iron Deposit Assessment

NCT01572922
View directory record Official record
Conditions
Iron Overload, Excessive Body Iron Burden
Interventions
R2*-UTE, R2*-GRE, Liver biopsy
Device · Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
142 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 11, 2019 · Synced Oct 10, 2026, 3:02 AM EDT
Completed Phase 2 Interventional Results available

Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

NCT00153985
View directory record Official record
Conditions
Hemoglobinopathies, Sickle Cell Disease, Thalassemia
Interventions
Busulfex, Fludarabine, Alemtuzumab, Stem Cell Transfusion
Drug · Procedure
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
6
States / cities
Atlanta, Georgia • Shreveport, Louisiana • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 30, 2013 · Synced Oct 10, 2026, 3:02 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

QST-Pupillometry in Sickle Cell Disease Patients

NCT02242058
View directory record Official record
Conditions
SCD With Severe Phenotype (HbSS, HbSβ0 Thalassemia, HbSOARab)
Interventions
Quantitative sensory testing
Other
Lead sponsor
Julia Finkel
Other
Eligibility
13 Years to 30 Years
Enrollment
96 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2018
U.S. locations
2
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 21, 2020 · Synced Oct 10, 2026, 3:02 AM EDT
Active, not recruiting No phase listed Observational

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062
View directory record Official record
Conditions
Blood and Lymphatic Diseases
Interventions
BIVV003, ST-400
Drug
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2038
U.S. locations
7
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 10, 2026, 3:02 AM EDT
Completed Not applicable Interventional

Use of Mobile Technology for Intensive Training in Medication Management

NCT02133560
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Video recording, Medication Administration Log, Education
Behavioral
Lead sponsor
Duke University
Other
Eligibility
5 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 25, 2017 · Synced Oct 10, 2026, 3:02 AM EDT
Recruiting Phase 2 Interventional

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

NCT02675959
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Defibrotide
Drug
Lead sponsor
New York Medical College
Other
Eligibility
6 Months to 21 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Valhalla, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 27, 2026 · Synced Oct 10, 2026, 3:02 AM EDT
Completed Phase 4 Interventional Results available

Pilot Study for Patients With Poor Response to Deferasirox

NCT00749515
View directory record Official record
Conditions
Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
Interventions
Deferoxamine, Deferasirox, HIDA
Drug · Radiation
Lead sponsor
Boston Children's Hospital
Other
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 12, 2024 · Synced Oct 10, 2026, 3:02 AM EDT
Terminated Phase 2 Interventional

Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

NCT00040469
View directory record Official record
Conditions
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
Interventions
Campath -1H, Dilantin, Busulfan, Cyclophosphamide
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Day to 64 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 18, 2020 · Synced Oct 10, 2026, 3:02 AM EDT
Completed No phase listed Observational

Study of Thiotepa and TEPA Drug Exposure in Pediatric Hematopoietic Stem Cell Transplant Patients

NCT03609840
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immune Deficiency, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Thiotepa
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Sep 21, 2023 · Synced Oct 10, 2026, 3:02 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

Fresh Versus Old Red Blood Cells for Transfusion

NCT01319552
View directory record Official record
Conditions
Iron, Abnormal Blood Level, Other Abnormal Blood Chemistry
Interventions
Fresh transfusion, Old transfusion
Procedure
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 65 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2010
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 2, 2015 · Synced Oct 10, 2026, 3:02 AM EDT
Not listed No phase listed Observational

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

NCT06539169
View directory record Official record
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Source: ClinicalTrials.gov public record
Updated Nov 14, 2024 · Synced Oct 10, 2026, 3:02 AM EDT
Available No phase listed Expanded access

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

NCT01881334
View directory record Official record
Conditions
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions
CliniMACS CD34 Reagent System
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 65 Years
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Oct 10, 2026, 3:02 AM EDT
Completed Phase 2 Interventional Results available

Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

NCT01186419
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-thalassemia
Interventions
SPD602 (FBS0701, SSP-004184)
Drug
Lead sponsor
Shire
Industry
Eligibility
18 Years to 60 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Oakland, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 10, 2021 · Synced Oct 10, 2026, 3:02 AM EDT