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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hemoglobinopathy; With Thalassemia Clear all
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 27, 2026 · Synced Jun 10, 2026, 2:24 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 20, 2017 · Synced Jun 10, 2026, 2:24 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 8, 2025 · Synced Jun 10, 2026, 2:24 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Combination Deferasirox and Deferiprone for Severe Iron Overload in Thalassemia

NCT01709032
View directory record Official record
Conditions
Thalassemia Major With Severe Transfusional Iron Overload
Interventions
Deferasirox and deferiprone
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
2
States / cities
Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 5, 2019 · Synced Jun 10, 2026, 2:24 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 19, 2023 · Synced Jun 10, 2026, 2:24 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

NCT02504619
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
CordIn
Biological
Lead sponsor
Gamida Cell ltd
Industry
Eligibility
2 Years to 25 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
2
States / cities
Oakland, California • Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jun 25, 2019 · Synced Jun 10, 2026, 2:24 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 Jun 10, 2026, 2:24 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 20, 2020 · Synced Jun 10, 2026, 2:24 AM EDT
Completed Not applicable Interventional

Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App

NCT04675645
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 25 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 28, 2021 · Synced Jun 10, 2026, 2:24 AM EDT
Completed Phase 2 Interventional

Phase II Study of Arginine Butyrate With or Without Epoetin Alfa in Patients With Thalassemia Intermedia

NCT00006136
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
arginine butyrate, epoetin alfa
Drug
Lead sponsor
Boston University
Other
Eligibility
3 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 1999
U.S. locations
6
States / cities
Oakland, California • New Haven, Connecticut • Bethesda, Maryland + 2 more
Source: ClinicalTrials.gov public record
Updated Nov 7, 2005 · Synced Jun 10, 2026, 2:24 AM EDT
Completed Phase 1 Interventional Results available

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

NCT00744692
View directory record Official record
Conditions
Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions
Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
Biological · Drug
Lead sponsor
Duke University
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Aug 12, 2014 · Synced Jun 10, 2026, 2:24 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 – 2027
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 11, 2025 · Synced Jun 10, 2026, 2:24 AM EDT
Completed Phase 1 Interventional

Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload

NCT01039636
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-thalassemia
Interventions
FBS0701
Drug
Lead sponsor
FerroKin BioSciences, Inc.
Industry
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2009
U.S. locations
3
States / cities
Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Feb 9, 2012 · Synced Jun 10, 2026, 2:24 AM EDT
Completed Phase 2 Interventional

Allogeneic Bone Marrow Transplantation for the Treatment of Genetic Disorders of Erythropoiesis

NCT00578435
View directory record Official record
Conditions
Genetic Disorders, Sickle Cell Anemia
Interventions
Busulfan, Cyclophosphamide, BMD
Procedure
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
1 Year and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1994 – 2008
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 11, 2008 · Synced Jun 10, 2026, 2:24 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 14, 2025 · Synced Jun 10, 2026, 2:24 AM EDT
Terminated Phase 2 Interventional

Bone Marrow Transplant From Donor Using Less Toxic Conditioning for Patient With High Risk Hemoglobinopathies

NCT00040417
View directory record Official record
Conditions
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
Interventions
FLUDARABINE, CAMPATH-IH, Total Body Irradiation, FK506, G-SCF (Granulocyte-colony stimulating factor)
Drug · Procedure
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Day to 65 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jan 17, 2020 · Synced Jun 10, 2026, 2:24 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Research Study of the Effect of Etavopivat on Other Drugs in Healthy Participants

NCT06813924
View directory record Official record
Conditions
Healthy Volunteers Sickle Cell Disease, Thalassemia
Interventions
Etavopivat, Digoxin, Pitavastatin, Metformin, Midazolam, Rosuvastatin
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
18 Years to 55 Years
Enrollment
37 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025
U.S. locations
1
States / cities
Salt Lake City, Utah
Source: ClinicalTrials.gov public record
Updated Sep 24, 2025 · Synced Jun 10, 2026, 2:24 AM EDT
Completed No phase listed Observational Results available

Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions

NCT00872833
View directory record Official record
Conditions
Thalassemia
Interventions
Not listed
Lead sponsor
Carelon Research
Other
Eligibility
18 Years and older
Enrollment
32 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
8
States / cities
Los Angeles, California • Oakland, California • Stanford, California + 5 more
Source: ClinicalTrials.gov public record
Updated Oct 6, 2015 · Synced Jun 10, 2026, 2:24 AM EDT
Recruiting Phase 2 Interventional

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

NCT06421636
View directory record Official record
Conditions
Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Interventions
REGN7999, Placebo
Drug
Lead sponsor
Regeneron Pharmaceuticals
Industry
Eligibility
18 Years to 65 Years
Enrollment
95 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced Jun 10, 2026, 2:24 AM EDT
Completed Phase 1Phase 2 Interventional Results available

HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

NCT02435901
View directory record Official record
Conditions
Sickle Cell Disease, Beta Thalassemia-Major
Interventions
alemtuzumab (Campath IH), Fludarabine, Melphalan, Cyclosporine, Mycophenolate mofetil, Tacrolimus, Hematopoietic Stem Cell Transplantation
Drug · Biological
Lead sponsor
Northwell Health
Other
Eligibility
1 Year to 21 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2019
U.S. locations
1
States / cities
New Hyde Park, New York
Source: ClinicalTrials.gov public record
Updated Aug 23, 2021 · Synced Jun 10, 2026, 2:24 AM EDT
Terminated No phase listed Observational

Amniotic Fluid Tandem Mass Spectrometry for Pregnancies Complicated by NIH and Severe Symmetrical IUGR

NCT00143039
View directory record Official record
Conditions
Hydrops Fetalis, Fetal Growth Retardation
Interventions
Tandem MS test for inborn errors of metabolism
Procedure
Lead sponsor
Obstetrix Medical Group
Industry
Eligibility
18 Years to 60 Years · Female only
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2009
U.S. locations
11
States / cities
Phoenix, Arizona • Laguna Hills, California • Long Beach, California + 6 more
Source: ClinicalTrials.gov public record
Updated Dec 18, 2014 · Synced Jun 10, 2026, 2:24 AM EDT
Completed No phase listed Observational

Bone Marrow for Hemoglobinopathy Research

NCT00669305
View directory record Official record
Conditions
Sickle Cell Anemia, Thalassemia
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Feb 24, 2020 · Synced Jun 10, 2026, 2:24 AM EDT
Recruiting Phase 1Phase 2 Interventional

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

NCT06364774
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
ALS20
Biological
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
18 Years to 40 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 14, 2026 · Synced Jun 10, 2026, 2:24 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 Jun 10, 2026, 2:24 AM EDT