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ClinicalTrials.gov public records Last synced Oct 13, 2026, 9:47 PM EDT

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Showing 121–144 of 208 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Hemoglobinopathy; With Thalassemia Clear all
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 13, 2026, 9:47 PM 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 13, 2026, 9:47 PM 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 13, 2026, 9:47 PM 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 13, 2026, 9:47 PM 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 13, 2026, 9:47 PM EDT
Completed Phase 3 Interventional Results available

An Extension Study of Iron Chelation Therapy With Deferasirox (ICL670) in β-thalassemia Patients With Transfusional Iron Overload

NCT00171210
View directory record Official record
Conditions
Transfusional Iron Overload in β-thalassemia
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
506 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2004
U.S. locations
6
States / cities
Los Angeles, California • Oakland, California • Stanford, California + 3 more
Source: ClinicalTrials.gov public record
Updated May 30, 2011 · Synced Oct 13, 2026, 9:47 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants

NCT03937817
View directory record Official record
Conditions
Alpha and Beta Thalassemia, Sickle Cell Disease, Malaria, Human Physiology
Interventions
Not listed
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years to 70 Years
Enrollment
300 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 2, 2026 · Synced Oct 13, 2026, 9:47 PM EDT
Completed Phase 2 Interventional Results available

Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia

NCT00661726
View directory record Official record
Conditions
Thalassemia
Interventions
Decitabine (USAN, INN)
Drug
Lead sponsor
Carelon Research
Other
Eligibility
18 Years and older
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
2
States / cities
Oakland, California • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 25, 2014 · Synced Oct 13, 2026, 9:47 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Multi-Center Study of Iron Overload: Pilot Study

NCT01114776
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
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2009 – 2013
U.S. locations
1
States / cities
Oakland, California
Source: ClinicalTrials.gov public record
Updated Sep 24, 2020 · Synced Oct 13, 2026, 9:47 PM EDT
Withdrawn Phase 2 Interventional

Allograft for Sickle Cell Disease and Thalassemia

NCT02038478
View directory record Official record
Conditions
Sickle Cell Disease and Thalassemia
Interventions
Donor Stem Cell Transplantation
Biological
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 29, 2021 · Synced Oct 13, 2026, 9:47 PM EDT
Terminated Phase 2 Interventional

A Study With LJPC-401 for the Treatment of Myocardial Iron Overload in Patients With Transfusion-Dependent Beta Thalassemia

NCT03381833
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LJPC-401
Drug
Lead sponsor
La Jolla Pharmaceutical Company
Industry
Eligibility
14 Years and older
Enrollment
84 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2020
U.S. locations
2
States / cities
Oakland, California • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 29, 2021 · Synced Oct 13, 2026, 9:47 PM EDT
Active, not recruiting Phase 2 Interventional Results available

A Study to Determine the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of AG-348 in Adult Participants With Non-transfusion-dependent Thalassemia

NCT03692052
View directory record Official record
Conditions
Thalassemia
Interventions
AG-348
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2030
U.S. locations
2
States / cities
Oakland, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 30, 2026 · Synced Oct 13, 2026, 9:47 PM 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 13, 2026, 9:47 PM EDT
Not listed Phase 1Phase 2 Interventional

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

NCT02061800
View directory record Official record
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 4, 2024 · Synced Oct 13, 2026, 9:47 PM EDT
Completed No phase listed Observational Results available

Assessment of Pain in People With Thalassemia

NCT00872339
View directory record Official record
Conditions
Thalassemia
Interventions
Not listed
Lead sponsor
Carelon Research
Other
Eligibility
12 Years to 90 Years
Enrollment
252 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
7
States / cities
Oakland, California • Atlanta, Georgia • Chicago, Illinois + 4 more
Source: ClinicalTrials.gov public record
Updated Jun 5, 2014 · Synced Oct 13, 2026, 9:47 PM 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 13, 2026, 9:47 PM 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 13, 2026, 9:47 PM EDT
Terminated Phase 2 Interventional Results available

Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere

NCT03825341
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Hydroxyurea, Hydroxyurea Oral Capsule
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
9 Months to 18 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 18, 2022 · Synced Oct 13, 2026, 9:47 PM EDT
Recruiting Phase 2 Interventional

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

NCT04099966
View directory record Official record
Conditions
Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Kostmann, Diamond Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Sickle Cell Disease, Beta-Thalassemia
Interventions
alpha beta depletion
Drug
Lead sponsor
Mitchell Cairo
Other
Eligibility
1 Day to 30 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Valhalla, New York
Source: ClinicalTrials.gov public record
Updated Aug 8, 2025 · Synced Oct 13, 2026, 9:47 PM EDT
Completed Phase 2 Interventional Results available

A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)

NCT03401112
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
IMR-687, Placebo
Drug
Lead sponsor
Cardurion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
7
States / cities
Oakland, California • Farmington, Connecticut • Hollywood, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 15, 2025 · Synced Oct 13, 2026, 9:47 PM EDT
Completed No phase listed Observational

Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)

NCT00000623
View directory record Official record
Conditions
Anemia, Cooley's, Beta-Thalassemia, Hematologic Diseases, Thalassemia, Osteoporosis, Iron Overload, Hypertension, Pulmonary
Interventions
Deferoxamine, Deferiprone, Arginine, Sildenafil, Decitabine
Drug
Lead sponsor
Carelon Research
Other
Eligibility
1 Year to 75 Years
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2006
U.S. locations
4
States / cities
Oakland, California • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 4, 2014 · Synced Oct 13, 2026, 9:47 PM EDT
Recruiting Phase 1 Interventional

Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

NCT01245179
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
panobinostat
Drug
Lead sponsor
Abdullah Kutlar
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2027
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 9, 2026 · Synced Oct 13, 2026, 9:47 PM EDT
Active, not recruiting Phase 1 Interventional

A Blood Stem Cell Transplant for Sickle Cell Disease

NCT03249831
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Disorder, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell
Interventions
Cyclophosphamide, Pentostatin, Rabbit anti-thymocyte globulin, Tacrolimus, Mycophenolate mofetil, CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant
Drug · Biological
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 45 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 13, 2026, 9:47 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study Evaluating the Safety and Efficacy of the LentiGlobin BB305 Drug Product in β-Thalassemia Major Participants

NCT01745120
View directory record Official record
Conditions
β-thalassemia Major
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
12 Years to 35 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
4
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated May 8, 2019 · Synced Oct 13, 2026, 9:47 PM EDT