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ClinicalTrials.gov public records Last synced Oct 10, 2026, 3:20 AM EDT

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Showing 97–120 of 208 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Hemoglobinopathy; With Thalassemia Clear all
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 10, 2026, 3:20 AM EDT
Active, not recruiting Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Non-Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-NTDT)

NCT04770753
View directory record Official record
Conditions
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Interventions
Placebo Matching Mitapivat, Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
194 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
6
States / cities
La Jolla, California • Palo Alto, California • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 28, 2026 · Synced Oct 10, 2026, 3:20 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 10, 2026, 3:20 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 10, 2026, 3:20 AM EDT
Terminated Not applicable Interventional Results available

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

NCT00578292
View directory record Official record
Conditions
Thalassemia
Interventions
Busulfan, Fludarabine, Campath 1H, Cyclophosphamide, MESNA
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
Up to 64 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2016
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated May 1, 2020 · Synced Oct 10, 2026, 3:20 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 18, 2026 · Synced Oct 10, 2026, 3:20 AM EDT
Completed Phase 3 Interventional Results available

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

NCT02604433
View directory record Official record
Conditions
Erythrocyte Transfusion, Beta-Thalassemia
Interventions
Luspatercept, Placebo
Drug · Other
Lead sponsor
Celgene
Industry
Eligibility
18 Years and older
Enrollment
336 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
6
States / cities
Los Angeles, California • Oakland, California • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 18, 2023 · Synced Oct 10, 2026, 3:20 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT)

NCT05444894
View directory record Official record
Conditions
Transfusion Dependent Beta Thalassemia, Hemoglobinopathies, Thalassemia Major, Thalassemia Intermedia
Interventions
EDIT-301
Genetic
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
18 Years to 35 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
7
States / cities
Oakland, California • Minneapolis, Minnesota • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2025 · Synced Oct 10, 2026, 3:20 AM EDT
Completed Phase 2 Interventional Results available

Phase II Study to Investigate the Benefits of an Improved Deferasirox Formulation (Film-coated Tablet)

NCT02125877
View directory record Official record
Conditions
Chronic Iron Overload Due to Transfusion-dependant Anemias
Interventions
Deferasirox dispersible tablet, Defearisox film-coated tablet
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
10 Years and older
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
Orange, California • Chicago, Illinois • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Jul 25, 2017 · Synced Oct 10, 2026, 3:20 AM 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 10, 2026, 3:20 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study

NCT00661804
View directory record Official record
Conditions
Thalassemia
Interventions
Not listed
Lead sponsor
Carelon Research
Other
Eligibility
5 Years and older
Enrollment
416 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2007 – 2011
U.S. locations
10
States / cities
Los Angeles, California • Oakland, California • Stanford, California + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2011 · Synced Oct 10, 2026, 3:20 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia

NCT05357482
View directory record Official record
Conditions
Sickle Cell Anemia, Beta Thalassemia
Interventions
TBI, Hydroxyurea, briquilimab, Filgrastim (G-CSF), Sirolimus, Alemtuzumab, Plerixafor
Radiation · Drug · Biological
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 100 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 10, 2026, 3:20 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional Results available

Haploidentical PBMC Transplant for Severe Congenital Anemias

NCT00977691
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
PBSC Transplant, Alemtuzumab, Sirolimus, Cyclophosphamide, Low Dose Irradiation
Procedure · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced Oct 10, 2026, 3:20 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 10, 2026, 3:20 AM EDT
Completed Phase 2Phase 3 Interventional

A Safety and Efficacy Study Evaluating CTX001 in Participants With Transfusion-Dependent β-Thalassemia

NCT03655678
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
12 Years to 35 Years
Enrollment
59 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
6
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced Oct 10, 2026, 3:20 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 10, 2026, 3:20 AM EDT
Completed Phase 2 Interventional Results available

Treosulfan and Fludarabine Phosphate Before Donor Stem Cell Transplant in Treating Patients With Nonmalignant Inherited Disorders

NCT00919503
View directory record Official record
Conditions
Non-Neoplastic Hematologic and Lymphocytic Disorder
Interventions
Allogeneic Bone Marrow Transplantation, Anti-Thymocyte Globulin, Cyclosporine, Fludarabine Phosphate, Laboratory Biomarker Analysis, Methotrexate, Mycophenolate Mofetil, Peripheral Blood Stem Cell Transplantation, Tacrolimus, Total-Body Irradiation, Treosulfan, Umbilical Cord Blood Transplantation
Procedure · Biological · Drug + 2 more
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
Up to 49 Years
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2020
U.S. locations
6
States / cities
Aurora, Colorado • Portland, Oregon • Nashville, Tennessee + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 13, 2021 · Synced Oct 10, 2026, 3:20 AM EDT
Completed Not applicable Interventional Results available

Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

NCT04523376
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia Major
Interventions
CliniMACS
Device
Lead sponsor
Timothy Olson
Other
Eligibility
2 Years to 25 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 28, 2026 · Synced Oct 10, 2026, 3:20 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 10, 2026, 3:20 AM EDT
Completed Phase 3 Interventional Results available

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

NCT00235391
View directory record Official record
Conditions
Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
Interventions
Deferasirox
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
2 Years and older
Enrollment
1,683 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2008
U.S. locations
27
States / cities
Little Rock, Arkansas • Berkeley, California • Orange, California + 23 more
Source: ClinicalTrials.gov public record
Updated Jun 7, 2011 · Synced Oct 10, 2026, 3:20 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 13, 2026 · Synced Oct 10, 2026, 3:20 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 10, 2026, 3:20 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 10, 2026, 3:20 AM 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 10, 2026, 3:20 AM EDT