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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Sickle Cell Disease and Thalassemia Clear all
Completed Phase 2 Interventional Results available

Phase II Randomized Trial:Arginine Butyrate Plus Standard Local Therapy in Patients With Refractory Sickle Cell Ulcers

NCT00004412
View directory record Official record
Conditions
Skin Ulcers, Sickle Cell Anemia
Interventions
Arginine Butyrate, Standard local care dressing
Drug · Other
Lead sponsor
Susan P. Perrine
Other
Eligibility
16 Years to 60 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1997 – 2005
U.S. locations
4
States / cities
Chicago, Illinois • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Mar 30, 2015 · Synced Jul 24, 2026, 4:14 AM EDT
Completed Phase 1Phase 2 Interventional Results available

Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation

NCT03513328
View directory record Official record
Conditions
Bone Marrow Failure Syndrome, Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Acquired Neutropenia in Newborn, Acquired Anemia Hemolytic, Acquired Thrombocytopenia, Hemophagocytic Lymphohistiocytoses, Wiskott-Aldrich Syndrome, Chronic Granulomatous Disease, Common Variable Immunodeficiency, X-linked Lymphoproliferative Disease, Severe Combined Immunodeficiency, Hurler Syndrome, Mannosidosis, Adrenoleukodystrophy
Interventions
Thiotepa--single daily dose, Thiotepa--escalated dose
Drug
Lead sponsor
University of Florida
Other
Eligibility
3 Months to 39 Years
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Gainesville, Florida
Source: ClinicalTrials.gov public record
Updated Sep 14, 2023 · Synced Jul 24, 2026, 4:14 AM EDT
Terminated Phase 2 Interventional

Bone Marrow Transplant From Related Donor for Patients With High Risk Hemoglobinopathies

NCT00040469
View directory record Official record
Conditions
Sickle Cell Anemia, Hemoglobinopathy, Thalassemia
Interventions
Campath -1H, Dilantin, Busulfan, Cyclophosphamide
Drug
Lead sponsor
Baylor College of Medicine
Other
Eligibility
1 Day to 64 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 Jul 24, 2026, 4:14 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 24, 2026, 4:14 AM EDT
Completed Not applicable Interventional Results available

A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen

NCT00427661
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Hemoglobinopathies
Interventions
Busulfan; Fludarabine; cyclosporine A and MMF
Other
Lead sponsor
University of Pittsburgh
Other
Eligibility
3 Years to 35 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2002 – 2014
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 17, 2016 · Synced Jul 24, 2026, 4:14 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 Jul 24, 2026, 4:14 AM EDT
Recruiting Phase 1 Interventional

Study of Panobinostat (LBH589) in Patients With Sickle Cell Disease

NCT01245179
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
panobinostat
Drug
Lead sponsor
Abdullah Kutlar
Other
Eligibility
18 Years and older
Enrollment
18 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2027
U.S. locations
1
States / cities
Augusta, Georgia
Source: ClinicalTrials.gov public record
Updated Jan 8, 2026 · Synced Jul 24, 2026, 4:14 AM 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 19, 2025 · Synced Jul 24, 2026, 4:14 AM EDT
Recruiting Phase 1Phase 2 Interventional

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

NCT07599176
View directory record Official record
Conditions
Sickle Cell Disease, Beta-thalassemia
Interventions
Total Body Irradiation, Alemtuzumab, Abatacept, Research blood draw
Radiation · Drug · Other
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
4 Years to 65 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Jul 24, 2026, 4:14 AM EDT
Not listed Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Improving the Results of Bone Marrow Transplantation for Patients With Severe Congenital Anemias

NCT00061568
View directory record Official record
Conditions
Congenital Hemolytic Anemia, Sickle Cell Disease
Interventions
Peripheral blood hematopoietic progenitor cell (PBPC) transplant, Alemtuzumab, Peripheral blood hematopoietic progenitor cell Apheresis, Sirolimus
Procedure · Drug
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
130 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2004 – 2026
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 28, 2024 · Synced Jul 24, 2026, 4:14 AM EDT
Recruiting Phase 2 Interventional

A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

NCT05736419
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Beta, Thalassemia
Interventions
Fludarabine, Cyclophosphamide, Tacrolimus, Mycophenolate Mofetil, Rabbit ATG, Dexamethasone, Bortezomib, Rituximab
Drug · Biological
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
2 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
6
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 3 more
Source: ClinicalTrials.gov public record
Updated Jun 2, 2026 · Synced Jul 24, 2026, 4:14 AM EDT
Active, not recruiting Phase 2 Interventional

An Extension Study of IMR-687 in Adult Patients With Sickle Cell Anemia

NCT04053803
View directory record Official record
Conditions
Sickle Cell Disease
Interventions
IMR-687
Drug
Lead sponsor
Imara, Inc.
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
Farmington, Connecticut • Hollywood, Florida • Temple, Texas
Source: ClinicalTrials.gov public record
Updated Mar 3, 2022 · Synced Jul 24, 2026, 4:14 AM 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 14, 2025 · Synced Jul 24, 2026, 4:14 AM EDT
Terminated Phase 2 Interventional Results available

Safety, Efficacy and Pharmacokinetics of an Oral Iron Chelator Given for a Year to Pediatric Patients With Iron Overload

NCT01363908
View directory record Official record
Conditions
Transfusional Iron Overload, Beta-Thalassemia
Interventions
SPD602
Drug
Lead sponsor
Shire
Industry
Eligibility
6 Years to 17 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
2
States / cities
Boston, Massachusetts • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jun 13, 2021 · Synced Jul 24, 2026, 4:14 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 24, 2026, 4:14 AM EDT
Terminated Not applicable Interventional Results available

Treatment of Sickle Cell Anemia With Stem Cell Transplant

NCT01350232
View directory record Official record
Conditions
Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease, Sickle Cell-β0-thalassemia
Interventions
Fludarabine, Cytarabine, Cellular Infusions, Total Body Irradiation, Cyclophosphamide, Bortezomib, Rituximab, Plasmapheresis
Drug · Device · Radiation + 1 more
Lead sponsor
Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
Other
Eligibility
18 Years to 45 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated May 15, 2025 · Synced Jul 24, 2026, 4:14 AM EDT
Recruiting Phase 1Phase 2 Interventional Accepts healthy volunteers

Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels

NCT04008368
View directory record Official record
Conditions
Myeloid Chimerism
Interventions
CliniMACS CD34 Reagent
Device
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
2 Years to 80 Years
Enrollment
32 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2037
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 4, 2026 · Synced Jul 24, 2026, 4:14 AM EDT
Completed Phase 2 Interventional Results available

The Effect of Rivaroxaban in Sickle Cell Disease

NCT02072668
View directory record Official record
Conditions
Sickle Cell Anemia, Sickle Cell-Beta0-Thalassemia
Interventions
rivaroxaban, placebo
Drug
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
18 Years to 65 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Apr 12, 2020 · Synced Jul 24, 2026, 4:14 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 Jul 24, 2026, 4:14 AM EDT
Completed Not applicable Interventional

Bone Marrow and Kidney Transplant for Patients With Chronic Kidney Disease and Blood Disorders

NCT01758042
View directory record Official record
Conditions
Chronic Kidney Disease, Acute Myeloid Leukemia (AML), Acute Lymphoblastic Leukemia (ALL), Chronic Myelogenous Leukemia (CML), Chronic Lymphocytic Leukemia (CLL), Non-Hodgkin's Lymphoma (NHL), Hodgkin Disease, Multiple Myeloma, Myelodysplastic Syndrome (MDS), Aplastic Anemia, AL Amyloidosis, Diamond Blackfan Anemia, Myelofibrosis, Myeloproliferative Disease, Sickle Cell Anemia, Autoimmune Diseases, Thalassemia
Interventions
Haploidentical Bone Marrow/Kidney
Procedure
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 70 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2025
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 5, 2026 · Synced Jul 24, 2026, 4:14 AM EDT
Completed Phase 1Phase 2 Interventional

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314
View directory record Official record
Conditions
Sickle Cell Disease, Thalassemia, Anemia, Granuloma, Wiskott-Aldrich Syndrome, Chediak Higashi Syndrome, Osteopetrosis, Neutropenia, Thrombocytopenia, Hurler Disease, Niemann-Pick Disease, Fucosidosis
Interventions
Hematopoietic stem cell transplantation
Procedure
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
Up to 21 Years
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2015
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jun 22, 2016 · Synced Jul 24, 2026, 4:14 AM EDT
Terminated Not applicable Interventional Results available

Bone Marrow Transplantation, Hemoglobinopathies, SCALLOP

NCT00578344
View directory record Official record
Conditions
Sickle Cell Disease, Hemoglobin SC
Interventions
Busulfan, Campath 1H, Cyclophosphamide and MESNA
Drug · Biological
Lead sponsor
Tami D. John
Other
Eligibility
Up to 40 Years
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2012
U.S. locations
2
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Jul 30, 2020 · Synced Jul 24, 2026, 4:14 AM EDT
Completed Phase 1 Interventional

Study to Determine the Maximum Tolerated Dose, Safety and Effectiveness of Pomalidomide for Patients With Sickle Cell Disease

NCT01522547
View directory record Official record
Conditions
Anemia, Sickle Cell
Interventions
pomalidomide
Drug
Lead sponsor
Celgene
Industry
Eligibility
18 Years to 60 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Detroit, Michigan
Source: ClinicalTrials.gov public record
Updated Nov 7, 2019 · Synced Jul 24, 2026, 4:14 AM EDT
Completed No phase listed Observational

Heart Disease in Sickle Cell Anemia

NCT00113152
View directory record Official record
Conditions
Sickle Cell Anemia, Pulmonary Hypertension
Interventions
Not listed
Lead sponsor
National Institutes of Health Clinical Center (CC)
NIH
Eligibility
18 Years to 60 Years
Enrollment
120 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2007
U.S. locations
2
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Jul 1, 2017 · Synced Jul 24, 2026, 4:14 AM EDT