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ClinicalTrials.gov public records Last synced Oct 6, 2026, 8:03 PM EDT

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Showing 49–72 of 132 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Sickle Cell Thalassemia Clear all
Terminated Phase 2 Interventional Results available

Hydroxyurea Therapy: Optimizing Access in Pediatric Populations Everywhere

NCT03825341
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Hydroxyurea, Hydroxyurea Oral Capsule
Drug
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
9 Months to 18 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 18, 2022 · Synced Oct 6, 2026, 8:03 PM EDT
Completed Phase 2 Interventional Results available

A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)

NCT03401112
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
IMR-687, Placebo
Drug
Lead sponsor
Cardurion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 55 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
7
States / cities
Oakland, California • Farmington, Connecticut • Hollywood, Florida + 3 more
Source: ClinicalTrials.gov public record
Updated May 15, 2025 · Synced Oct 6, 2026, 8:03 PM 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 6, 2026, 8:03 PM 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 3, 2025 · Synced Oct 6, 2026, 8:03 PM EDT
Terminated Phase 2 Interventional

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases

NCT03367546
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, High Risk Hematologic Disorders, Cerebral Adrenoleukodystrophy, Inherited Metabolic Disorders
Interventions
Blood and Marrow Transplant
Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
0 Years to 25 Years
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 12, 2024 · Synced Oct 6, 2026, 8:03 PM EDT
Completed Phase 1 Interventional

Topical Sodium Nitrite for Chronic Leg Ulcers in Adult Patients With Blood Disorders

NCT01316796
View directory record Official record
Conditions
Sickle Cell Anemia, Sickle Cell Disease, Chronic Hemolytic Disorders
Interventions
Sodium Nitrite Cream
Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 99 Years
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 22, 2018 · Synced Oct 6, 2026, 8:03 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

NCT05635266
View directory record Official record
Conditions
Age-Related Macular Degeneration, Allergies, Alpha-Gal Syndrome, Alzheimer Disease, Amyloidosis, Ankylosing Spondylitis, Arthritis, Alopecia Areata, Asthma, Atopic Dermatitis, Autism, Autoimmune Hepatitis, Behcet's Disease, Beta-Thalassemia, Cancer, Celiac Disease, Kidney Diseases, COPD, Crohn Disease, Cystic Fibrosis, Diabetes, Dravet Syndrome, DMD, Fibromyalgia, Graves Disease, Thyroid Diseases, Hepatitis, Hidradenitis Suppurativa, ITP, Leukemia, ALS, Lupus or SLE, Lymphoma, Multiple Sclerosis, Myasthenia Gravis, Heart Diseases, Parkinson Disease, Pemphigus Vulgaris, Cirrhosis, Psoriasis, Schizophrenia, Scleroderma, Sickle Cell Disease, Stroke, Ulcerative Colitis, Vasculitis, Vitiligo
Interventions
Specimen sample
Diagnostic Test
Lead sponsor
Sanguine Biosciences
Industry
Eligibility
18 Years to 85 Years
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2025
U.S. locations
1
States / cities
Waltham, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 7, 2024 · Synced Oct 6, 2026, 8:03 PM 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 10, 2012 · Synced Oct 6, 2026, 8:03 PM EDT
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 2, 2023 · Synced Oct 6, 2026, 8:03 PM EDT
Completed Phase 1 Interventional

The Role of Endothelin-1 in Sickle Cell Disease

NCT02712346
View directory record Official record
Conditions
Sickle Cell Anemia
Interventions
Ambrisentan, Placebo
Drug
Lead sponsor
Augusta University
Other
Eligibility
18 Years to 65 Years
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Apr 2, 2020 · Synced Oct 6, 2026, 8:03 PM EDT
Active, not recruiting Phase 2 Interventional

A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001)

NCT05353647
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 40 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
9
States / cities
Los Angeles, California • Oakland, California • Sacramento, California + 4 more
Source: ClinicalTrials.gov public record
Updated Aug 25, 2026 · Synced Oct 6, 2026, 8:03 PM 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 21, 2017 · Synced Oct 6, 2026, 8:03 PM EDT
Active, not recruiting Phase 1 Interventional

A Blood Stem Cell Transplant for Sickle Cell Disease

NCT03249831
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Disorder, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell
Interventions
Cyclophosphamide, Pentostatin, Rabbit anti-thymocyte globulin, Tacrolimus, Mycophenolate mofetil, CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant
Drug · Biological
Lead sponsor
City of Hope Medical Center
Other
Eligibility
18 Years to 45 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
1
States / cities
Duarte, California
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Oct 6, 2026, 8:03 PM EDT
Recruiting Phase 3 Interventional

A Research Study Looking at Long-term Treatment With Etavopivat in People With Sickle Cell Disease or Thalassaemia

NCT06609226
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Etavopivat A, Etavopivat B, Etavopivat C
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
2 Years and older
Enrollment
480 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
36
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 27 more
Source: ClinicalTrials.gov public record
Updated Sep 18, 2026 · Synced Oct 6, 2026, 8:03 PM 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 6, 2026, 8:03 PM EDT
Available No phase listed Expanded access

Expanded Access to T-cell Depleted Haplo-Identical Stem Cells for Patients Receiving Haplo-Identical and Unrelated Cord Blood Transplants

NCT01881334
View directory record Official record
Conditions
Hematologic Malignancies, Inborn Errors of Metabolism Disorders, Immune Deficiencies
Interventions
CliniMACS CD34 Reagent System
Biological
Lead sponsor
Joanne Kurtzberg, MD
Other
Eligibility
Up to 65 Years
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced Oct 6, 2026, 8:03 PM 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 18, 2020 · Synced Oct 6, 2026, 8:03 PM 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 6, 2026, 8:03 PM EDT
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Hemoglobinopathy

NCT00176852
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Severe Congenital Neutropenia, Diamond-Blackfan Anemia, Shwachman-Diamond Syndrome
Interventions
Busulfan, Fludarabine, ATG, TLI, Busulfan, Cyclophosphamide, ATG, GCSF, Campath, Fludarabine, Cyclophosphamide, Total Body Irradiation, Stem cell infusion
Drug · Radiation · Procedure
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 50 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 27, 2020 · Synced Oct 6, 2026, 8:03 PM EDT
Completed No phase listed Observational

Multi-Center Study of Iron Overload: Survey Study (MCSIO)

NCT01913548
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
423 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
6
States / cities
Oakland, California • Augusta, Georgia • Chicago, Illinois + 3 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2020 · Synced Oct 6, 2026, 8:03 PM 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 4, 2008 · Synced Oct 6, 2026, 8:03 PM 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 15, 2025 · Synced Oct 6, 2026, 8:03 PM EDT
Completed 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
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
1
States / cities
Chicago, Illinois
Source: ClinicalTrials.gov public record
Updated Jul 31, 2026 · Synced Oct 6, 2026, 8:03 PM EDT
Completed Not applicable Interventional Accepts healthy volunteers Results available

PINPOINT: Gaming Technology for SCD Pain

NCT04579926
View directory record Official record
Conditions
Sickle Cell Disease, Sickle Cell Anemia in Children, Sickle Cell Thalassemia, Sickle Cell SC Disease
Interventions
Pinpoint app
Other
Lead sponsor
Klein Buendel, Inc.
Industry
Eligibility
13 Years to 17 Years
Enrollment
24 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2022
U.S. locations
2
States / cities
Golden, Colorado • Munster, Indiana
Source: ClinicalTrials.gov public record
Updated May 1, 2024 · Synced Oct 6, 2026, 8:03 PM EDT