Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 5, 2026, 7:36 PM EDT

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Showing 25–48 of 75 matching trials from the live ClinicalTrials.gov search.
Conditions
Sickle Cell Disease
Interventions
Conditioning Regimen with Bone Marrow Transplant
Drug
Lead sponsor
Emory University
Other
Eligibility
16 Years to 40 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
8
States / cities
Oakland, California • Washington D.C., District of Columbia • Miami, Florida + 4 more
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
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, 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
Conditions
Sickle Cell Disease
Interventions
αβ+ T-cell depletion with Miltenyi CliniMACS system
Device
Lead sponsor
University of Chicago
Other
Eligibility
2 Years to 25 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
1
States / cities
Chicago, Illinois
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
Conditions
Bone Marrow Failure Syndrome, Severe Aplastic Anemia, Severe Congenital Neutropenia, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Schwachman Diamond Syndrome, Primary Immunodeficiency Syndromes, Acquired Immunodeficiency Syndromes, Histiocytic Syndrome, Familial Hemophagocytic Lymphocytosis, Lymphohistiocytosis, Macrophage Activation Syndrome, Langerhans Cell Histiocytosis (LCH), Hemoglobinopathies, Sickle Cell Disease, Sickle Cell-beta-thalassemia
Interventions
CD34 Stem Cell Selection Therapy
Biological
Lead sponsor
Diane George
Other
Eligibility
Up to 40 Years
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2030
U.S. locations
1
States / cities
New York, New York
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
Conditions
Sickle Cell Disease, Hematological Diseases, Hemoglobinopathies
Interventions
Exa-cel
Biological
Lead sponsor
Vertex Pharmaceuticals Incorporated
Industry
Eligibility
12 Years to 35 Years
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
9
States / cities
Palo Alto, California • Chicago, Illinois • New York, New York + 4 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
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
Conditions
Anemia, Sickle Cell
Interventions
Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years to 45 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2021
U.S. locations
1
States / cities
Duarte, California
Conditions
Non Malignant Disorders, Immunodeficiencies, Congenital Marrow Failures, Hemoglobinopathies, Inborn Errors of Metabolism, Sickle Cell, Thalassemia, Lysosomal Storage Disease
Interventions
Unrelated Umbilical Cord Blood Transplant, Reduced Intensity Conditioning
Biological · Drug
Lead sponsor
Duke University
Other
Eligibility
Up to 21 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2014
U.S. locations
1
States / cities
Durham, North Carolina
Conditions
Chronic Myeloid Leukemia (CML), Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS), Juvenile Myelomonocytic Leukemia (JMML), Acute Lymphoblastic Leukemia (ALL), Lymphoma (Hodgkin's and Non-Hodgkin's)
Interventions
CliniMACS CD34+ Reagent System, Thiotepa, Cyclophosphamide, Alemtuzumab, Tacrolimus, Melphalan, Busulfan, Fludarabine, Methylprednisolone
Device · Drug
Lead sponsor
Diane George
Other
Eligibility
Up to 22 Years
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2026
U.S. locations
1
States / cities
New York, New York
Conditions
Mortality in Sickle Cell, Sickle Cell Cardiopulmonary Complications, Sickle Cell Organ Damage, Sickle Cell Life Expectancy and Risk Factors for Early Death, Sickle Cell Lung Disease and Sudden Death
Interventions
Not listed
Lead sponsor
National Heart, Lung, and Blood Institute (NHLBI)
NIH
Eligibility
18 Years to 100 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2035
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Sickle Cell Disease
Interventions
Quality of Life measures
Behavioral
Lead sponsor
Emory University
Other
Eligibility
3 Years to 23 Years
Enrollment
33 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2005 – 2014
U.S. locations
2
States / cities
Atlanta, Georgia
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
Conditions
Sickle Cell Disease
Interventions
Alemtuzumab, Total Body Irradiation, Sirolimus
Drug · Radiation
Lead sponsor
Kathleen Dorritie
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Conditions
Graft Versus Host Disease
Interventions
Abatacept
Drug
Lead sponsor
Emory University
Other
Eligibility
Up to 20 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
9
States / cities
Birmingham, Alabama • Wilmington, Delaware • Atlanta, Georgia + 6 more
Conditions
Sickle Cell Disease
Interventions
CRISPR_SCD001
Drug
Lead sponsor
Mark Walters, MD
Other
Eligibility
12 Years to 35 Years
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
2
States / cities
Los Angeles, California • Oakland, California
Conditions
Sickle Cell Disease, Beta Thalassemia-Major
Interventions
alemtuzumab (Campath IH), Fludarabine, Melphalan, Cyclosporine, Mycophenolate mofetil, Tacrolimus, Hematopoietic Stem Cell Transplantation
Drug · Biological
Lead sponsor
Northwell Health
Other
Eligibility
1 Year to 21 Years
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2019
U.S. locations
1
States / cities
New Hyde Park, New York
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
Conditions
Sickling Disorder Due to Hemoglobin S
Interventions
131I-apamistmab, Sirolimus, Campath, Total Body Irradiation, Exchange Transfusion, Planar gamma imaging
Drug · Radiation · Procedure
Lead sponsor
Columbia University
Other
Eligibility
12 Years to 50 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
New York, New York
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