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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Thalassemia Clear all
Completed Phase 2 Interventional Results available

Cardiac T2* in Beta-thalassemia Patients on Deferasirox Treatment

NCT00447694
View directory record Official record
Conditions
Beta-thalassemia, Iron Overload
Interventions
Deferasirox
Drug
Lead sponsor
Novartis
Industry
Eligibility
10 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
3
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Jul 21, 2026, 7:12 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 12, 2026 · Synced Jul 21, 2026, 7:12 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 22, 2017 · Synced Jul 21, 2026, 7:12 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Siplizumab for Sickle Cell Disease Transplant

NCT06078696
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
Siplizumab, Exchange Transfusion, Total Body Irradiation, Stem Cell Infusion, Cyclophosphamide 50mg, Mesna, Sirolimus Oral Tablet, Rituximab or Biosimilar
Drug · Procedure
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 50 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 9, 2026 · Synced Jul 21, 2026, 7:12 AM EDT
Completed Phase 2 Interventional

Pilot Study to Assess the Safety, PK and Iron Chelating Activity of DST-0509 (Deferasirox) in Thalassemia Patients Refractory to Chelation

NCT03637556
View directory record Official record
Conditions
Thalassemia Major
Interventions
DST-0509, Jadenu, Exjade
Drug
Lead sponsor
DisperSol Technologies, LLC
Industry
Eligibility
8 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 31, 2022 · Synced Jul 21, 2026, 7:12 AM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

NCT02906202
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 17, 2023 · Synced Jul 21, 2026, 7:12 AM EDT
Not listed Phase 1Phase 2 Interventional

Iron Balance Study of Deferasirox, Deferoxamine and the Combination of Both

NCT00738413
View directory record Official record
Conditions
Thalassemia
Interventions
Deferoxamine, Deferasirox
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
18 Years to 50 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2009
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 4, 2010 · Synced Jul 21, 2026, 7:12 AM EDT
Completed No phase listed Observational

Study of Fludarabine Drug Exposure in Pediatric Bone Marrow Transplantation

NCT01316549
View directory record Official record
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Fludarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Oct 3, 2021 · Synced Jul 21, 2026, 7:12 AM EDT
Completed Phase 2 Interventional Results available

L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia

NCT00125788
View directory record Official record
Conditions
Sickle Cell Anemia, Thalassemia
Interventions
L-glutamine, Placebo
Drug
Lead sponsor
Emmaus Medical, Inc.
Industry
Eligibility
5 Years and older
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2008
U.S. locations
5
States / cities
Bellflower, California • Torrance, California • Atlanta, Georgia + 2 more
Source: ClinicalTrials.gov public record
Updated Jan 28, 2021 · Synced Jul 21, 2026, 7:12 AM EDT
Active, not recruiting Phase 2 Interventional Accepts healthy volunteers Results available

Nonmyeloablative Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease and Beta-thalassemia in People With Higher Risk of Transplant Failure

NCT02105766
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Stem Cell Transplantation, Graft vs Host Disease
Interventions
Alemtuzumab, Sirolimus, Cyclophosphamide, Pentostatin, Radiotherapy, Filgrastim
Drug · Procedure
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 80 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 10, 2025 · Synced Jul 21, 2026, 7:12 AM EDT
Completed No phase listed Observational

Multi-Center Study of Iron Overload: Survey Study (MCSIO)

NCT01913548
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Diamond-Blackfan Anemia
Interventions
Not listed
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
16 Years and older
Enrollment
423 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
6
States / cities
Oakland, California • Augusta, Georgia • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 22, 2020 · Synced Jul 21, 2026, 7:12 AM EDT
Enrolling by invitation Phase 3 Interventional

A Long-term Follow-up Study in Participants Who Received CTX001

NCT04208529
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2039
U.S. locations
9
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 5 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced Jul 21, 2026, 7:12 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 26, 2025 · Synced Jul 21, 2026, 7:12 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 10, 2025 · Synced Jul 21, 2026, 7:12 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 28, 2016 · Synced Jul 21, 2026, 7:12 AM EDT
Completed Phase 2 Interventional Results available

Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload

NCT00901199
View directory record Official record
Conditions
Thalassemia, Iron Overload
Interventions
Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
Drug
Lead sponsor
Elliott Vichinsky
Other
Eligibility
8 Years and older
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2012
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Aug 11, 2021 · Synced Jul 21, 2026, 7:12 AM EDT
Not listed Phase 2 Interventional

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

NCT00408447
View directory record Official record
Conditions
Sickle Cell Disease, Beta Thalassemia
Interventions
Busulfan, Fludarabine, Alemtuzumab, Allogeneic stem cell transplant
Drug · Procedure
Lead sponsor
Columbia University
Other
Eligibility
1 Month to 30 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2025
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 12, 2024 · Synced Jul 21, 2026, 7:12 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 9, 2012 · Synced Jul 21, 2026, 7:12 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 2, 2026 · Synced Jul 21, 2026, 7:12 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 24, 2017 · Synced Jul 21, 2026, 7:12 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 8, 2011 · Synced Jul 21, 2026, 7:12 AM EDT
Terminated Phase 2 Interventional Results available

Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

NCT01363908
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-Thalassemia
Interventions
SPD602
Drug
Lead sponsor
Shire
Industry
Eligibility
6 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Jul 21, 2026, 7:12 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 19, 2022 · Synced Jul 21, 2026, 7:12 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 17, 2026 · Synced Jul 21, 2026, 7:12 AM EDT