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Showing 1–24 of 62 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Transfusion-dependent Anemia Clear all
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 7, 2026, 10:16 PM 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 2, 2026 · Synced Sep 7, 2026, 10:16 PM EDT
Terminated Phase 2 Interventional Results available

Deferasirox in Treating Patients With Very Low, Low, or Intermediate-Risk Red Blood Cell Transfusion Dependent Anemia or Myelodysplastic Syndrome

NCT02943668
View directory record Official record
Conditions
Anemia, Myelodysplastic Syndrome
Interventions
Deferasirox, Laboratory Biomarker Analysis
Drug · Other
Lead sponsor
Fred Hutchinson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2018
U.S. locations
1
States / cities
Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Jun 30, 2020 · Synced Sep 7, 2026, 10:16 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 4, 2014 · Synced Sep 7, 2026, 10:16 PM 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 7, 2026, 10:16 PM EDT
Not yet recruiting Not applicable Interventional

Hypoxic Red Blood Cells in Sickle Cell Anemia

NCT06743113
View directory record Official record
Conditions
Sickle Cell Anaemia, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Interventions
Hemanext ONE System, Conventional RBCs
Device
Lead sponsor
Hemanext
Industry
Eligibility
7 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2029
U.S. locations
5
States / cities
Farmington, Connecticut • Atlanta, Georgia • Chicago, Illinois + 2 more
Source: ClinicalTrials.gov public record
Updated Aug 16, 2026 · Synced Sep 7, 2026, 10:16 PM EDT
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 7, 2026, 10:16 PM EDT
Enrolling by invitation No phase listed Observational

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

NCT06363760
View directory record Official record
Conditions
Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia, Hemoglobinopathies
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Editas Medicine, Inc.
Industry
Eligibility
12 Years to 50 Years
Enrollment
54 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2040
U.S. locations
16
States / cities
Oakland, California • Aurora, Colorado • New Haven, Connecticut + 12 more
Source: ClinicalTrials.gov public record
Updated Apr 1, 2025 · Synced Sep 7, 2026, 10:16 PM 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 17, 2023 · Synced Sep 7, 2026, 10:16 PM EDT
Recruiting Phase 1 Interventional

Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia

NCT07476183
View directory record Official record
Conditions
RPS19 Deficient Diamond-Blackfan Anemia
Interventions
APR-2020
Biological
Lead sponsor
Apriligen, Inc.
Industry
Eligibility
2 Years to 25 Years
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
Stanford, California • Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Jul 20, 2026 · Synced Sep 7, 2026, 10:16 PM 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 7, 2026, 10:16 PM 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 7, 2026, 10:16 PM EDT
Active, not recruiting Phase 3 Interventional

A Study to Assess Luspatercept in Lower-risk Myelodysplastic Syndrome Participants

NCT06045689
View directory record Official record
Conditions
Myelodysplastic Syndromes
Interventions
Luspatercept
Drug
Lead sponsor
Bristol-Myers Squibb
Industry
Eligibility
18 Years and older
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
16
States / cities
Los Alamitos, California • New Haven, Connecticut • St. Petersburg, Florida + 13 more
Source: ClinicalTrials.gov public record
Updated Jun 14, 2026 · Synced Sep 7, 2026, 10:16 PM 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 7, 2026, 10:16 PM EDT
Completed Phase 2 Interventional

Leucovorin for the Treatment of 5 q Minus Syndrome

NCT00004997
View directory record Official record
Conditions
5q Minus Syndrome, Myelodysplastic Syndrome
Interventions
Leucovorin
Drug
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
Not listed
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1998 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Sep 7, 2026, 10:16 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 28, 2021 · Synced Sep 7, 2026, 10:16 PM 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 7, 2026, 10:16 PM EDT
Completed Phase 1Phase 2 Interventional Results available

Pilot Phase I/II Study of Amino Acid Leucine in Treatment of Patients With Transfusion-Dependent Diamond Blackfan Anemia

NCT01362595
View directory record Official record
Conditions
Diamond Blackfan Anemia, Blackfan Diamond Syndrome, DBA, Congenital Hypoplastic Anemia, Pure Red Cell Aplasia
Interventions
leucine
Drug
Lead sponsor
Northwell Health
Other
Eligibility
2 Years and older
Enrollment
55 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2020
U.S. locations
11
States / cities
Phoenix, Arizona • Palo Alto, California • Indianapolis, Indiana + 8 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2022 · Synced Sep 7, 2026, 10:16 PM EDT
Terminated Phase 4 Interventional Results available

Physiologic Effects of RBC Transfusion

NCT02566577
View directory record Official record
Conditions
Red Blood Cell Transfusion
Interventions
Fresh red blood cell (RBC) transfusion, Storage-aged red blood cell (RBC) transfusion, Electronic infusion pump
Biological · Device
Lead sponsor
Emory University
Other
Eligibility
21 Years to 80 Years
Enrollment
15 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Atlanta, Georgia
Source: ClinicalTrials.gov public record
Updated May 23, 2019 · Synced Sep 7, 2026, 10:16 PM EDT
Not yet recruiting Phase 1Phase 2 Interventional

A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes

NCT07712562
View directory record Official record
Conditions
Bone Marrow Failure Syndromes, Aplastic Anemias, Severe Aplastic Anemia (SAA), Severe Aplastic Anemia, Relapse, Severe Aplastic Anemia, Refractory, Non-severe Aplastic Anemia
Interventions
Dibotatug (DR-01)
Drug
Lead sponsor
Dren Bio
Industry
Eligibility
18 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
10
States / cities
Duarte, California • Palo Alto, California • Miami, Florida + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 23, 2026 · Synced Sep 7, 2026, 10:16 PM 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 Sep 7, 2026, 10:16 PM EDT
Completed Phase 2 Interventional

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia

NCT03802201
View directory record Official record
Conditions
β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
Interventions
PTG-300
Drug
Lead sponsor
Protagonist Therapeutics, Inc.
Industry
Eligibility
12 Years to 65 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
3
States / cities
Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 15, 2021 · Synced Sep 7, 2026, 10:16 PM EDT
Completed Phase 3 Interventional

Study Using Eculizumab in Transfusion Dependent Paroxysmal Nocturnal Hemoglobinuria (PNH) Patients

NCT00122330
View directory record Official record
Conditions
Hemoglobinuria, Paroxysmal
Interventions
eculizumab
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
75 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
16
States / cities
Duarte, California • Los Angeles, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Dec 3, 2006 · Synced Sep 7, 2026, 10:16 PM 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 7, 2026, 10:16 PM EDT