Independent directory Public ClinicalTrials.gov records United States
Clear filters

These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.

ClinicalTrials.gov public records Last synced Oct 22, 2026, 2:36 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 132 matching trials from the live ClinicalTrials.gov search.
Conditions
Thalassemia, Sickle Cell Disease, Healthy Controls
Interventions
Hydroxyurea, Acetazolamide
Drug
Lead sponsor
Children's Hospital Los Angeles
Other
Eligibility
7 Years and older
Enrollment
165 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2022
U.S. locations
1
States / cities
Los Angeles, California
Conditions
Beta-Thalassemia, Thalassemia, Sickle Cell Disease, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Sickle Cell Anemia
Interventions
CTX001
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
2 Years and older
Enrollment
160 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2039
U.S. locations
9
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 5 more
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
Conditions
Transfusional Iron Overload, Beta-thalassemia
Interventions
SPD602 (FBS0701, SSP-004184)
Drug
Lead sponsor
Shire
Industry
Eligibility
18 Years to 60 Years
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
2
States / cities
Oakland, California • Boston, Massachusetts
Conditions
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Acute Myeloid Leukemia With FLT3/ITD Mutation, Acute Myeloid Leukemia With Gene Mutations, Aplastic Anemia, B-Cell Non-Hodgkin Lymphoma, CD40 Ligand Deficiency, Chronic Granulomatous Disease, Chronic Leukemia in Remission, Chronic Lymphocytic Leukemia, Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Chronic Myelomonocytic Leukemia, Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Congenital Amegakaryocytic Thrombocytopenia, Congenital Neutropenia, Congenital Pure Red Cell Aplasia, Glanzmann Thrombasthenia, Immunodeficiency Syndrome, Myelodysplastic Syndrome, Myelofibrosis, Myeloproliferative Neoplasm, Paroxysmal Nocturnal Hemoglobinuria, Plasma Cell Myeloma, Polycythemia Vera, Recurrent Non-Hodgkin Lymphoma, Refractory Non-Hodgkin Lymphoma, Secondary Acute Myeloid Leukemia, Secondary Myelodysplastic Syndrome, Severe Aplastic Anemia, Shwachman-Diamond Syndrome, Sickle Cell Disease, T-Cell Non-Hodgkin Lymphoma, Thalassemia, Waldenstrom Macroglobulinemia, Wiskott-Aldrich Syndrome
Interventions
Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis, Peripheral Blood Stem Cell Transplantation, Total-Body Irradiation
Drug · Other · Procedure + 1 more
Lead sponsor
Roswell Park Cancer Institute
Other
Eligibility
1 Year to 75 Years
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
1
States / cities
Buffalo, New York
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
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
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
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
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
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Hemoglobinopathy

NCT00176852
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
Terminated Phase 1Phase 2 Interventional Results available

Allogeneic SCT of CordIn™, in Patients With Hemoglobinopathies

NCT02504619
Conditions
Sickle Cell Disease, Thalassemia
Interventions
CordIn
Biological
Lead sponsor
Gamida Cell ltd
Industry
Eligibility
2 Years to 25 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2017
U.S. locations
2
States / cities
Oakland, California • Washington D.C., District of Columbia
Conditions
Sickle Cell Disease, HEMOGLOBIN SS, Hemoglobin S Beta-0 Thalassemia, Inflammation, Quality of Life
Interventions
Omega-3 Fatty Acids: Eicosapentaenoic Acid (EPA) and Docosahexaenoic Acid (DHA), Placebo Capsules
Dietary Supplement · Other
Lead sponsor
Thomas Jefferson University
Other
Eligibility
10 Years to 19 Years
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2012
U.S. locations
2
States / cities
Philadelphia, Pennsylvania
Conditions
Transfusion Dependent Beta Thalassemia, Sickle Cell Disease
Interventions
Not listed
Lead sponsor
Georgetown University
Other
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
Sickle Cell Anemia, Thalassemia
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2019
U.S. locations
1
States / cities
Memphis, Tennessee
Conditions
Sickle Cell Disease, Sickle-Beta Zero Thalassemia
Interventions
SCD-101
Drug
Lead sponsor
Invenux, LLC
Industry
Eligibility
18 Years to 55 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2023
U.S. locations
1
States / cities
Brooklyn, New York
Conditions
Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis, Creutzfeld-Jakob Disease, Cystic Fibrosis, Duchenne Muscular Dystrophy, Early-Onset Alzheimer Disease, Ehlers-Danlos Syndrome, Huntington Disease, Gaucher Disease, GM1 Gangliosidosis, Myasthenia Gravis, Pompe Disease, Sickle Cell Disease, Transthyretin Amyloid Cardiomyopathy, Rare Diseases
Interventions
Not listed
Lead sponsor
xCures
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Los Altos, California
Conditions
Sickle Cell Disease, Thalassemia
Interventions
Video recording, Medication Administration Log, Education
Behavioral
Lead sponsor
Duke University
Other
Eligibility
5 Years and older
Enrollment
46 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
1
States / cities
Durham, North Carolina
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
Completed Not applicable Interventional Accepts healthy volunteers Results available

Fresh Versus Old Red Blood Cells for Transfusion

NCT01319552
Conditions
Iron, Abnormal Blood Level, Other Abnormal Blood Chemistry
Interventions
Fresh transfusion, Old transfusion
Procedure
Lead sponsor
Columbia University
Other
Eligibility
18 Years to 65 Years
Enrollment
14 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2008 – 2010
U.S. locations
1
States / cities
New York, New York
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
Conditions
Sickle Cell Disease and Thalassemia
Interventions
Donor Stem Cell Transplantation
Biological
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
18 Years to 45 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
1
States / cities
Dallas, Texas
Conditions
Sickle Cell-thalassemia Disease, Thalassemia
Interventions
peripheral blood stem cell graft that are CD34+ selected
Drug
Lead sponsor
Catherine Bollard
Other
Eligibility
Up to 22 Years
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2024
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Conditions
Sickle Cell Disease
Interventions
Defibrotide
Drug
Lead sponsor
New York Medical College
Other
Eligibility
6 Months to 21 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2027
U.S. locations
4
States / cities
Los Angeles, California • Gainesville, Florida • Valhalla, New York + 1 more