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ClinicalTrials.gov public records Last synced Oct 7, 2026, 2:21 AM EDT

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Showing 25–48 of 208 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Hemoglobinopathy; With Thalassemia Clear all
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 7, 2026, 2:21 AM EDT
Terminated Phase 1 Interventional

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

NCT02986698
View directory record Official record
Conditions
Alpha Thalassemia Major, Hemoglobinopathy; With Thalassemia, Hemoglobinopathies, Fetal Anemia, Fetal Hydrops, Alpha; Thalassemia, Thalassemia Major, Thalassemia Alpha, A-Thalassemia
Interventions
in utero hematopoietic stem cell transplantation
Biological
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Weeks to 26 Weeks
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 27, 2025 · Synced Oct 7, 2026, 2:21 AM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

NCT01419704
View directory record Official record
Conditions
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Up to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 3, 2023 · Synced Oct 7, 2026, 2:21 AM EDT
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Effects of Transfusion of Older Stored Red Cells

NCT01534676
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Transfusion, Blood, Chelation therapy
Procedure · Biological
Lead sponsor
Columbia University
Other
Eligibility
1 Year to 65 Years
Enrollment
3 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2013
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Oct 5, 2016 · Synced Oct 7, 2026, 2:21 AM EDT
Completed No phase listed Observational

Heart Disease in Sickle Cell Anemia

NCT00113152
View directory record Official record
Conditions
Sickle Cell Anemia, Pulmonary Hypertension
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years to 60 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 2, 2017 · Synced Oct 7, 2026, 2:21 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 7, 2026, 2:21 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 7, 2026, 2:21 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 7, 2026, 2:21 AM EDT
Active, not recruiting Phase 2 Interventional

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

NCT06421636
View directory record Official record
Conditions
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Interventions
REGN7999, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 7, 2026, 2:21 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 7, 2026, 2:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Quantitative Susceptibility Mapping (QSM) to Guide Iron Chelating Therapy

NCT04171635
View directory record Official record
Conditions
MRI Scans
Interventions
Quantitative Susceptibility Mapping (QSM) Magnetic Resonance Imaging (MRI), R2* Magnetic Resonance Imaging (MRI)
Radiation
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
2 Years and older
Enrollment
55 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2025
U.S. locations
2
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 27, 2026 · Synced Oct 7, 2026, 2:21 AM EDT
Terminated Phase 2 Interventional

Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia

NCT00034528
View directory record Official record
Conditions
Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia, Thalassemia Major
Interventions
Busulfan, Fludarabine, FK506, Prednisone
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 1, 2013 · Synced Oct 7, 2026, 2:21 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 7, 2026, 2:21 AM EDT
Completed No phase listed Observational

Long Term Outcomes in β Thalassemia Major

NCT02307786
View directory record Official record
Conditions
Thalassemia Major
Interventions
Not listed
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
0 Years to 30 Years
Enrollment
176 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Feb 11, 2025 · Synced Oct 7, 2026, 2:21 AM EDT
Completed Phase 2 Interventional

Evaluating the Safety of Two Medications to Treat Hepatitis C in People With Thalassemia (The HepC Study)

NCT00502788
View directory record Official record
Conditions
Hepatitis C, Thalassemia
Interventions
Peginterferon Alfa-2a and Ribavirin, Ribavirin
Drug
Lead sponsor
Carelon Research
Other
Eligibility
18 Years to 44 Years
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2006
U.S. locations
3
States / cities
Oakland, California • New York, New York • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 4, 2014 · Synced Oct 7, 2026, 2:21 AM EDT
Completed Phase 2 Interventional

Cord Blood Transplantation for Sickle Cell Anemia and Thalassemia

NCT00029380
View directory record Official record
Conditions
Hematologic Diseases, Anemia, Sickle Cell, Beta-Thalassemia, Hematopoietic Stem Cell Transplantation
Interventions
Sangstat, Cyclophosphamide, Busulfan, Mycophenolate Mofetil, Cyclosporine, Cord Blood Transplantation
Drug · Procedure
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
3 Years to 14 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2006
U.S. locations
14
States / cities
Oakland, California • Washington D.C., District of Columbia • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2016 · Synced Oct 7, 2026, 2:21 AM EDT
Completed No phase listed Observational

Study of Clofarabine and Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation (HCT)

NCT03609814
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi's Anemia, Thalassemia, Sickle Cell Disease
Interventions
Clofarabine, Fludarabine Injection
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2020
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 4, 2021 · Synced Oct 7, 2026, 2:21 AM EDT
Completed Phase 2 Interventional

A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

NCT01044186
View directory record Official record
Conditions
Transfusional Iron Overload
Interventions
ICL670
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2003
U.S. locations
8
States / cities
Oakland, California • Orange, California • Boston, Massachusetts + 4 more
Source: ClinicalTrials.gov public record
Updated Feb 23, 2017 · Synced Oct 7, 2026, 2:21 AM EDT
Recruiting No phase listed Observational

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

NCT06271512
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
No Intervention
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
Not listed
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2043
U.S. locations
6
States / cities
Oakland, California • Palo Alto, California • Minneapolis, Minnesota + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 11, 2025 · Synced Oct 7, 2026, 2:21 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

High-Tc Susceptometer to Monitor Transfusional Iron Overload

NCT01241357
View directory record Official record
Conditions
Transfusional Iron Overload, Thalassemia Major, Sickle Cell Disease, Myelodysplastic Syndromes, Aplastic Anemia
Interventions
Hepatic biomagnetic susceptibility measurement
Device
Lead sponsor
Columbia University
Other
Eligibility
5 Years to 80 Years
Enrollment
99 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2016
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 21, 2016 · Synced Oct 7, 2026, 2:21 AM EDT
Completed Phase 1 Interventional

Cardiopulmonary Function Assessment and NO-Based Therapies for Patients With Hemolysis-Associated Pulmonary Hypertension

NCT00352430
View directory record Official record
Conditions
Pulmonary Hypertension
Interventions
Nitric Oxide/INP Pulse Delivery, Nitric Oxide/INO Pulse Delivery
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2010
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 25, 2019 · Synced Oct 7, 2026, 2:21 AM EDT
Completed Phase 2 Interventional Results available

Extension Study of Iron Chelation Therapy With Deferasirox in β-thalassemia and Rare Chronic Anemia Patients

NCT00303329
View directory record Official record
Conditions
Anemia, Hemosiderosis
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
184 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
5
States / cities
Oakland, California • Stanford, California • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated May 9, 2011 · Synced Oct 7, 2026, 2:21 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 7, 2026, 2:21 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 7, 2026, 2:21 AM EDT