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ClinicalTrials.gov public records Last synced Oct 2, 2026, 8:03 AM EDT

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Showing 1–24 of 68 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Sickle B+ 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, 8:03 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, 8:03 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, 8:03 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, 8:03 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, 8:03 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, 8:03 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, 8:03 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, 8:03 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, 8:03 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, 8:03 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 Aug 18, 2026 · Synced Oct 2, 2026, 8:03 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 2, 2026, 8:03 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

NCT00061568
View directory record Official record
Conditions
Congenital Hemolytic Anemia, Sickle Cell Disease
Interventions
Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis, Sirolimus
Procedure · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2028
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Sep 9, 2026 · Synced Oct 2, 2026, 8:03 AM EDT
Completed Not applicable Interventional

Study of Allogeneic Bone Marrow Transplantation Using Matched, Related Donors in Patients With Nonmalignant Hematologic Disorders

NCT00005893
View directory record Official record
Conditions
Neutropenia, Sickle Cell Anemia, Thalassemia Major, Red-Cell Aplasia, Pure
Interventions
anti-thymocyte globulin, busulfan, cyclophosphamide, Bone marrow transplantation
Drug · Procedure
Lead sponsor
Fairview University Medical Center
Other
Eligibility
0 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2000
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 24, 2005 · Synced Oct 2, 2026, 8:03 AM EDT
Recruiting Phase 3 Interventional

Evaluation of Efficacy and Safety of a Single Dose of CTX001 in Participants With Transfusion-Dependent β-Thalassemia and Severe Sickle Cell Disease

NCT05477563
View directory record Official record
Conditions
Beta-Thalassemia, Thalassemia, Hematologic Diseases, Genetic Diseases, Inborn, Hemoglobinopathies, Sickle Cell Disease, Sickle Cell Anemia
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
3
States / cities
New York, New York • Charlotte, North Carolina • Nashville, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 23, 2026 · Synced Oct 2, 2026, 8:03 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 2, 2026, 8:03 AM EDT
Recruiting Phase 1 Interventional

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

NCT06647979
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
Interventions
autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452
Biological · Device
Lead sponsor
Daniel Bauer
Other
Eligibility
13 Years to 40 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 3, 2026 · Synced Oct 2, 2026, 8:03 AM EDT
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

NCT04008368
View directory record Official record
Conditions
Myeloid Chimerism
Interventions
CliniMACS CD34 Reagent
Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2037
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 5, 2026 · Synced Oct 2, 2026, 8:03 AM EDT
Completed Phase 1 Interventional

Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease

NCT01522547
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
pomalidomide
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 8, 2019 · Synced Oct 2, 2026, 8:03 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 2, 2026, 8:03 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 2, 2026, 8:03 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 2, 2026, 8:03 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 14, 2021 · Synced Oct 2, 2026, 8:03 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 2, 2026, 8:03 AM EDT