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Showing 1–24 of 29 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Hemoglobin Beta Thalassemia Clear all
Completed Not applicable Interventional

Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators

NCT02197845
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Patient Navigator, Recruitment into Specialty Care
Behavioral
Lead sponsor
Virginia Commonwealth University
Other
Eligibility
15 Years and older
Enrollment
353 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2018
U.S. locations
1
States / cities
Richmond, Virginia
Source: ClinicalTrials.gov public record
Updated Jun 1, 2023 · Synced Jul 22, 2026, 5:50 AM EDT
Completed Phase 1 Interventional

A Phase I/II Trial of Recombinant-Methionyl Human Stem Cell Factor (SCF) in Adult Patients With Sickling Disorders

NCT00005783
View directory record Official record
Conditions
Hemoglobin SC Disease, Sickle Cell Anemia
Interventions
Recombinant-methionyl human stem cell factor
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 22, 2026, 5:50 AM 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 Jul 22, 2026, 5:50 AM EDT
Recruiting Phase 2 Interventional

MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies

NCT05735717
View directory record Official record
Conditions
Hematologic Malignancy, Acute Leukemia, Remission, Acute Myeloid Leukemia, Acute Lymphoblastic Leukemia, AML, TP53, Intrachromosomal Amplification of Chromosome 21, Cytogenetic Abnormality, CNS Leukemia, Minimal Residual Disease, Myelodysplasia, Juvenile Myelomonocytic Leukemia, Somatic Mutation, PTPN11 Gene Mutation, N-RAS Gene Amplification, Neurofibromatosis 1, NF1 Mutation, CBL Gene Mutation, Monosomy 7, Chromosome Abnormality, Fetal Hemoglobin, Lymphoblastic Lymphoma, High Grade Non-Hodgkin's Lymphoma, Adult
Interventions
Fludarabine, Busulfan, Melphalan, Rituximab, Levetiracetam, Alpha/Beta T Cell-Depleted Hematopoietic Stem Cells, Thymoglobulin, Cyclophosphamide
Drug · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 60 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Apr 5, 2026 · Synced Jul 22, 2026, 5:50 AM EDT
Completed Phase 2 Interventional

Hydroxyurea to Treat Beta-Thalassemia (Cooley's Anemia)

NCT00001958
View directory record Official record
Conditions
Beta Thalassemia, Hemoglobinopathy
Interventions
Hydroxyurea
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1999 – 2002
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 22, 2026, 5:50 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 Jul 22, 2026, 5:50 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 Jul 22, 2026, 5:50 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 Jul 22, 2026, 5:50 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 17, 2026 · Synced Jul 22, 2026, 5:50 AM EDT
Enrolling by invitation Phase 2 Interventional

Open-Label Extension Study to Pioneer Study 6058-SCD-101

NCT07401823
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
Pociredir
Drug
Lead sponsor
Fulcrum Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
8
States / cities
Little Rock, Arkansas • Los Angeles, California • Baton Rouge, Louisiana + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2026 · Synced Jul 22, 2026, 5:50 AM EDT
Recruiting Not applicable Interventional

An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease

NCT04688411
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Beta Zero Thalassemia, Sickle B+ Thalassemia, Sickle Cell Hemoglobin C
Interventions
MED-Go App
Behavioral
Lead sponsor
Ann & Robert H Lurie Children's Hospital of Chicago
Other
Eligibility
12 Years to 21 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Nov 19, 2024 · Synced Jul 22, 2026, 5:50 AM EDT
Terminated Phase 2 Interventional

Stem Cell Transplantation After Reduced-Dose Chemotherapy for Patients With Sickle Cell Disease or Thalassemia

NCT00034528
View directory record Official record
Conditions
Hemoglobinopathies, Anemia, Sickle Cell, Hemoglobin SC Disease, Thalassemia, Thalassemia Major
Interventions
Busulfan, Fludarabine, FK506, Prednisone
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2001 – 2003
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 30, 2013 · Synced Jul 22, 2026, 5:50 AM EDT
Active, not recruiting 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 Dec 23, 2024 · Synced Jul 22, 2026, 5:50 AM EDT
Active, not recruiting No phase listed Observational

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

NCT05675436
View directory record Official record
Conditions
Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss, Hbsc, Sickle Beta Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Syndrome Variant
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
16 Years to 80 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 22, 2026, 5:50 AM EDT
Completed Phase 2 Interventional

5-Azacytidine and Phenylbutyrate to Treat Severe Thalassemia

NCT00005934
View directory record Official record
Conditions
Beta Thalassemia
Interventions
5-Azacytidine
Drug
Lead sponsor
National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
NIH
Eligibility
Not listed
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2003
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 3, 2008 · Synced Jul 22, 2026, 5:50 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 8, 2018 · Synced Jul 22, 2026, 5:50 AM EDT
Completed Phase 1 Interventional Accepts healthy volunteers

A Study of IMR-687 in Healthy Adult Volunteers

NCT02998450
View directory record Official record
Conditions
Sickle Cell Disease, Sickle-Cell; Hb-SC, Sickle Beta 0 Thalassemia
Interventions
IMR-687, Placebo Oral Capsule
Drug
Lead sponsor
Cardurion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
66 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2016 – 2017
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated May 14, 2025 · Synced Jul 22, 2026, 5:50 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 Jul 22, 2026, 5:50 AM EDT
Recruiting Phase 1 Interventional Accepts healthy volunteers

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease

NCT05904093
View directory record Official record
Conditions
Sickle Cell Disease, Hb-SS Disease, Hemoglobin S, Disease Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin Beta Thalassemia Disease
Interventions
Fostamatinib
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 65 Years
Enrollment
25 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 6, 2026 · Synced Jul 22, 2026, 5:50 AM 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 7, 2019 · Synced Jul 22, 2026, 5:50 AM EDT
Completed Not applicable Interventional Accepts healthy volunteers

Effect of Broccoli Sprouts Homogenate on SS RBC

NCT01715480
View directory record Official record
Conditions
Sickle Red Blood Cell, Fetal Hemoglobin, Oxidative Stress
Interventions
Broccosprouts® (Brassica Protection Products LLC) homogenate
Dietary Supplement
Lead sponsor
Duke University
Other
Eligibility
18 Years and older
Enrollment
21 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2015
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated May 27, 2015 · Synced Jul 22, 2026, 5:50 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 Jul 22, 2026, 5:50 AM EDT
Completed Phase 2 Interventional Results available

Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies

NCT00153985
View directory record Official record
Conditions
Hemoglobinopathies, Sickle Cell Disease, Thalassemia
Interventions
Busulfex, Fludarabine, Alemtuzumab, Stem Cell Transfusion
Drug · Procedure
Lead sponsor
Dana-Farber Cancer Institute
Other
Eligibility
18 Years and older
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2009
U.S. locations
6
States / cities
Atlanta, Georgia • Shreveport, Louisiana • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 29, 2013 · Synced Jul 22, 2026, 5:50 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 10, 2026 · Synced Jul 22, 2026, 5:50 AM EDT