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Showing 1–24 of 35 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Transfusion Dependent Beta Thalassemia Clear all
Completed Phase 2 Interventional

Safety Study for Beta Thalassemia Subjects on PTG-300

NCT04054921
View directory record Official record
Conditions
β-thalassemia, Ineffective Erythropoiesis
Interventions
PTG-300
Drug
Lead sponsor
Protagonist Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
34 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
2
States / cities
Oakland, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 15, 2021 · Synced Sep 29, 2026, 1:59 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 12, 2026 · Synced Sep 29, 2026, 1:59 AM EDT
Completed Phase 4 Interventional Results available

Pilot Study for Patients With Poor Response to Deferasirox

NCT00749515
View directory record Official record
Conditions
Transfusion-dependent Hemachromatosis, Thalassemia Major, Sickle Cell Disease
Interventions
Deferoxamine, Deferasirox, HIDA
Drug · Radiation
Lead sponsor
Boston Children's Hospital
Other
Eligibility
6 Years and older
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 11, 2024 · Synced Sep 29, 2026, 1:59 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 Sep 22, 2026 · Synced Sep 29, 2026, 1:59 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

The BENeFiTS Trial in Beta Thalassemia Intermedia

NCT04432623
View directory record Official record
Conditions
Beta Thalassemia Intermedia, Sickle Cell Disease
Interventions
Benserazide Only Product
Drug
Lead sponsor
Phoenicia BioScience
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
4
States / cities
Oakland, California • Boston, Massachusetts • Weston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Aug 14, 2025 · Synced Sep 29, 2026, 1:59 AM EDT
Completed Phase 2 Interventional Results available

Efficacy and Safety of Deferasirox in Non-transfusion Dependent Thalassemia Patients With Iron Overload and a One Year Open-label Extension Study

NCT00873041
View directory record Official record
Conditions
Non-transfusion Dependent Thalassemia
Interventions
deferasirox, placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
10 Years and older
Enrollment
166 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2012
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 8, 2013 · Synced Sep 29, 2026, 1:59 AM EDT
Active, not recruiting No phase listed Observational

Long - Term Follow Up of Sickle Cell Disease and Beta-thalassemia Subjects Previously Exposed to BIVV003 or ST-400.

NCT05145062
View directory record Official record
Conditions
Blood and Lymphatic Diseases
Interventions
BIVV003, ST-400
Drug
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 45 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2038
U.S. locations
7
States / cities
Oakland, California • Sacramento, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 11, 2025 · Synced Sep 29, 2026, 1:59 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 16, 2025 · Synced Sep 29, 2026, 1:59 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 Sep 29, 2026, 1:59 AM EDT
Withdrawn Phase 2 Interventional

Efficacy and Safety Study of Multiple Doses of VIT-2763 in Adults With Transfusion-dependent Beta-thalassemia

NCT04938635
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
VIT-2763 60 mg QD, VIT-2763 60 mg BID, VIT-2763 120 mg BID, Placebo
Drug
Lead sponsor
Vifor (International) Inc.
Industry
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
1
States / cities
Whittier, California
Source: ClinicalTrials.gov public record
Updated Nov 9, 2022 · Synced Sep 29, 2026, 1:59 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 1, 2025 · Synced Sep 29, 2026, 1:59 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 Apr 30, 2020 · Synced Sep 29, 2026, 1:59 AM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia, Who do Not Have a β0/β0 Genotype

NCT02906202
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 17, 2023 · Synced Sep 29, 2026, 1:59 AM EDT
Terminated Phase 1 Interventional

Safety and Pharmacokinetic Study of Escalating Doses of SP-420, an Iron Chelator, in Patients With β-Thalassemia

NCT02274233
View directory record Official record
Conditions
Iron Overload, Beta-Thalassemia
Interventions
SP-420
Drug
Lead sponsor
Sideris Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2015
U.S. locations
3
States / cities
Boston, Massachusetts • New York, New York • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 28, 2015 · Synced Sep 29, 2026, 1:59 AM EDT
Completed Phase 1Phase 2 Interventional Results available

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

NCT03432364
View directory record Official record
Conditions
Transfusion Dependent Beta-thalassemia
Interventions
ST-400 Investigational product
Genetic
Lead sponsor
Sangamo Therapeutics
Industry
Eligibility
18 Years to 40 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
6
States / cities
Los Angeles, California • Oakland, California • Atlanta, Georgia + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 13, 2023 · Synced Sep 29, 2026, 1:59 AM EDT
Recruiting Phase 3 Interventional

A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia

NCT07517133
View directory record Official record
Conditions
Non-Transfusion-dependent Alpha-Thalassemia, Non-Transfusion-dependent Beta-Thalassemia
Interventions
Mitapivat, Placebo Matching Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
1 Year to 17 Years
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2032
U.S. locations
5
States / cities
Palo Alto, California • Atlanta, Georgia • New York, New York + 2 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2026 · Synced Sep 29, 2026, 1:59 AM EDT
Completed Phase 2 Interventional

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

NCT04987489
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Etavopivat tablets
Drug
Lead sponsor
Forma Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
14
States / cities
Cerritos, California • Los Angeles, California • Oakland, California + 9 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2025 · Synced Sep 29, 2026, 1:59 AM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/II Pilot Study of Mixed Chimerism to Treat Hemoglobinopathies

NCT01419704
View directory record Official record
Conditions
Anemia, Sickle Cell, Complex and Transfusion-dependent Hemoglobinopathies, Thalassemia, Diamond-Blackfan Anemia, Bone Marrow Failure Syndromes, Alpha-Thalassemia, Beta-Thalassemia
Interventions
Enriched Hematopoetic Stem Cell Infusion
Biological
Lead sponsor
Talaris Therapeutics Inc.
Industry
Eligibility
Up to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
3
States / cities
Chicago, Illinois • Louisville, Kentucky • Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Mar 2, 2023 · Synced Sep 29, 2026, 1:59 AM EDT
Active, not recruiting Phase 3 Interventional Results available

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

NCT04770779
View directory record Official record
Conditions
Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
Interventions
Placebo Matching Mitapivat, Mitapivat
Drug
Lead sponsor
Agios Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
258 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2029
U.S. locations
10
States / cities
Phoenix, Arizona • La Jolla, California • Oakland, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 27, 2026 · Synced Sep 29, 2026, 1:59 AM 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 28, 2021 · Synced Sep 29, 2026, 1:59 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 Sep 29, 2026, 1:59 AM EDT
Completed Phase 3 Interventional Results available

A Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Participants With Transfusion-Dependent β-Thalassemia

NCT03207009
View directory record Official record
Conditions
Beta-Thalassemia
Interventions
LentiGlobin BB305 Drug Product
Genetic
Lead sponsor
Genetix Biotherapeutics Inc.
Industry
Eligibility
0 Years to 50 Years
Enrollment
19 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
3
States / cities
Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 6, 2024 · Synced Sep 29, 2026, 1:59 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 22, 2026 · Synced Sep 29, 2026, 1:59 AM EDT
Terminated Not applicable Interventional

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

NCT02179359
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion Dependent Alpha- or Beta- Thalassemia, Diamond Blackfan Anemia, Paroxysmal Nocturnal Hemoglobinuria, Glanzmann Thrombasthenia, Severe Congenital Neutropenia, Shwachman-Diamond Syndrome, Non-Malignant Hematologic Disorders
Interventions
Reduced Toxicity Ablative Regimen, Reduced Intensity Preparative Regimen, Myeloablative Preparative Regimen
Drug
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 55 Years
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Mar 2, 2025 · Synced Sep 29, 2026, 1:59 AM EDT