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ClinicalTrials.gov public records Last synced Oct 2, 2026, 7:41 AM EDT

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Showing 1–24 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 2, 2026, 7:41 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 2, 2026, 7:41 AM EDT
Active, not recruiting No phase listed Observational

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

NCT02633943
View directory record Official record
Conditions
Transfusion-dependent Beta-Thalassemia
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2035
U.S. locations
6
States / cities
Oakland, California • Chicago, Illinois • Bethesda, Maryland + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 9, 2025 · Synced Oct 2, 2026, 7:41 AM EDT
Recruiting Phase 2 Interventional

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415
View directory record Official record
Conditions
Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions
Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa
Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
2 Months to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Oct 2, 2026, 7:41 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 2, 2026, 7:41 AM EDT
Enrolling by invitation No phase listed Observational

Decision-Making and Quality of Life Surrounding Hematologic Disease and Gene Therapy

NCT06664541
View directory record Official record
Conditions
Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Georgetown University
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Oct 29, 2024 · Synced Oct 2, 2026, 7:41 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension

NCT00872170
View directory record Official record
Conditions
Thalassemia, Hypertension, Pulmonary
Interventions
Sildenafil
Drug
Lead sponsor
Carelon Research
Other
Eligibility
7 Years and older
Enrollment
27 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Feb 21, 2014 · Synced Oct 2, 2026, 7:41 AM EDT
Completed Phase 1Phase 2 Interventional

Iron Balance Study of DFO and GT56-252 in Patients With Transfusional Iron Overload Secondary to Beta-Thalassemia

NCT00069862
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
Deferitrin (GT56-252), desferoxamine (DFO)
Drug
Lead sponsor
Genzyme, a Sanofi Company
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 5, 2015 · Synced Oct 2, 2026, 7:41 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 2, 2026, 7:41 AM EDT
Completed Phase 2 Interventional

5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia

NCT00005934
View directory record Official record
Conditions
Beta Thalassemia
Interventions
5-Azacytidine
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 2, 2026, 7:41 AM EDT
Terminated Not applicable Interventional Results available

Treatment of Sickle Cell Anemia With Stem Cell Transplant

NCT01350232
View directory record Official record
Conditions
Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease, Sickle Cell-β0-thalassemia
Interventions
Fludarabine, Cytarabine, Cellular Infusions, Total Body Irradiation, Cyclophosphamide, Bortezomib, Rituximab, Plasmapheresis
Drug · Device · Radiation + 1 more
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Other
Eligibility
18 Years to 45 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 16, 2025 · Synced Oct 2, 2026, 7:41 AM EDT
Recruiting Phase 1Phase 2 Interventional

T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias

NCT03653338
View directory record Official record
Conditions
Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia
Interventions
CD3/CD19 depleted leukocytes, CD45RA depleted leukocytes, Hydroxyurea, Rituximab, Alemtuzumab, Fludarabine, Thiotepa
Biological · Drug
Lead sponsor
Paul Szabolcs
Other
Eligibility
5 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2028
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 13, 2026 · Synced Oct 2, 2026, 7:41 AM EDT
Temporarily Not Available No phase listed Expanded access

Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease

NCT07640815
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
Biological
Lead sponsor
David Williams
Other
Eligibility
13 Years to 55 Years
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jun 11, 2026 · Synced Oct 2, 2026, 7:41 AM EDT
Completed Phase 2 Interventional Results available

A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

NCT03342404
View directory record Official record
Conditions
Thalassemia
Interventions
Luspatercept, Placebo, Best Supportive Care (BSC)
Drug · Other
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older
Enrollment
145 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Dec 20, 2023 · Synced Oct 2, 2026, 7:41 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 2, 2026, 7:41 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 2, 2026, 7:41 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 2, 2026, 7:41 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 2, 2026, 7:41 AM EDT
Completed Not applicable Interventional

An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease

NCT04688411
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
Interventions
MED-Go App
Behavioral
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 21 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 2, 2026, 7:41 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Reproductive Capacity and Iron Burden in Thalassemia

NCT02308904
View directory record Official record
Conditions
THALASSEMIA MAJOR
Interventions
Blood Draw/Semen Exam, Retrospective data/Chart Review/Relevant Clinical Results, Pituitary MRI
Other
Lead sponsor
UCSF Benioff Children's Hospital Oakland
Other
Eligibility
12 Years and older
Enrollment
59 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2020
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Dec 23, 2020 · Synced Oct 2, 2026, 7:41 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Research Study of the Effect of Food on Etavopivat in Healthy Participants

NCT06433661
View directory record Official record
Conditions
Healthy Volunteers Sickle Cell Disease, Thalassemia
Interventions
Etavopivat
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 55 Years
Enrollment
16 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Oct 10, 2025 · Synced Oct 2, 2026, 7:41 AM EDT
Active, not recruiting Phase 3 Interventional

Evaluation of Safety and Efficacy of CTX001 in Pediatric Participants With Transfusion-Dependent β-Thalassemia (TDT)

NCT05356195
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Genetic Diseases, Inborn, Hematologic Diseases, Hemoglobinopathies
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years to 11 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Jul 1, 2026 · Synced Oct 2, 2026, 7:41 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 2, 2026, 7:41 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 2, 2026, 7:41 AM EDT