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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

Conditions: Transfusional Iron Overload, Thalassemia Major, Sickle Cell Disease, Myelodysplastic Syndromes, Aplastic Anemia
Interventions: Hepatic biomagnetic susceptibility measurement; Device
Lead sponsor: Columbia University; Other
Eligibility: 5 Years to 80 Years
Enrollment: 99 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2016
U.S. locations: 1
States / cities: New York, New York

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: 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

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

Conditions: Acute Leukemia, Severe Aplastic Anemia, Non-hodgkin Lymphoma, Hodgkin Lymphoma, Kostmann, Diamond Blackfan Anemia, Amegakaryocytic Thrombocytopenia, Sickle Cell Disease, Beta-Thalassemia
Interventions: alpha beta depletion; Drug
Lead sponsor: Mitchell Cairo; Other
Eligibility: 1 Day to 30 Years
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2027
U.S. locations: 1
States / cities: Valhalla, New York

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

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: Sickle Cell Disease, Beta Thalassemia
Interventions: Busulfan, Fludarabine, Alemtuzumab, Allogeneic stem cell transplant; Drug · Procedure
Lead sponsor: Columbia University; Other
Eligibility: 1 Month to 30 Years
Enrollment: 53 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2025
U.S. locations: 1
States / cities: New York, New York

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

Conditions: Sickle Cell Disease, Thalassemia, Beta, Thalassemia Alpha, Hemoglobinopathies
Interventions: GWAS; Genetic
Lead sponsor: Cyprus Institute of Neurology and Genetics; Other
Eligibility: 2 Years and older
Enrollment: 30,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0), Beta-Thalassemia, Transfusion Dependent Beta-Thalassaemia
Interventions: autologous bone marrow derived CD34+ HSPCs electroporated with BCL11A enhancer targeting Cas9 ribonucleoprotein, Sequencing Assay for Variant rs114518452; Biological · Device
Lead sponsor: Daniel Bauer; Other
Eligibility: 13 Years to 40 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Sickle Cell Disease
Interventions: Defibrotide; Drug
Lead sponsor: New York Medical College; Other
Eligibility: 6 Months to 34 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

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

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

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: All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis, Asthma, Atopic Dermatitis Eczema, Beta Thalassemia, Bipolar Disorder, Breast Cancer, Celiac Disease, Cervical Cancer, Chronic Inflammatory Demyelinating Polyneuropathy, Chronic Kidney Diseases, Chronic Obstructive Pulmonary Disease, Colon Cancer, Colorectal Cancer, Crohn's Disease, Cystic Fibrosis, Depression, Diabetes Mellitus, Duchenne Muscular Dystrophy, Endometriosis, Epilepsy, Facioscapulohumeral Muscular Dystrophy, G6PD Deficiency, General Anxiety Disorder, Hepatitis B, Hereditary Hemorrhagic Telangiectasia, HIV/AIDS, Human Papilloma Virus, Huntington's Disease, Idiopathic Thrombocytopenic Purpura, Insomnia, Kidney Cancer, Leukemia, Lung Cancer, Lupus Nephritis, Lymphoma, Melanoma, Multiple Myeloma, Multiple Sclerosis, Myositis, Myotonic Dystrophy, Ovarian Cancer, Pancreatic Cancer, Parkinson's Disease, Polycystic Kidney Diseases, Prostate Cancer, Psoriasis, Psoriatic Arthritis, Rosacea, Scleroderma, Sickle Cell Anemia, Sickle Cell Trait, Sjogren's Syndrome, Skin Cancer, Spinal Muscular Atrophy, Systemic Lupus Erythematosus, Thrombotic Thrombocytopenic Purpura, Trisomy 21, Ulcerative Colitis
Interventions: Not listed
Lead sponsor: Sanguine Biosciences; Industry
Eligibility: 18 Years to 100 Years
Enrollment: 17,667 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013 – 2024
U.S. locations: 1
States / cities: Los Angeles, California

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, 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

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

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: 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: 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

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

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Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease

High-Tc Susceptometer to Monitor Transfusional Iron Overload

CD34+ (Malignant) Stem Cell Selection for Patients Receiving Allogenic Stem Cell Transplant

Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies

A Study of Etavopivat in Patients With Thalassemia or Sickle Cell Disease

AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion

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

Enhancing Use of Hydroxyurea In Sickle Cell Disease Using Patient Navigators

Stem Cell Transplant in Sickle Cell Disease and Thalassemia

Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

Study of the Role of Genetic Modifiers in Hemoglobinopathies

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Severe β-Hemoglobinopathies

Myeloablative Conditioning, Prophylactic Defibrotide and Haplo AlloSCT for Patients With Sickle Cell Disease

Hematopoietic Stem Cell Transplant for High Risk Hemoglobinopathies

Home-based Assessment of PRO Measures in SCD Using A Smartphone App Platform: A Feasibility Study

Safety and Pharmacodynamic Study of an Oral Iron Chelator Given for 6 Months to Patients With Iron Overload

At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Partial Stem Cell Transplant for Sickle Cell Disease From Matched Donors

Adherence to HU and HRQOL in Patients With Sickle Cell Disease: An Intervention Study Using HU-Go App

HSCT For Patients With High Risk Hemoglobinopathies Using Reduced Intensity

Dose-Escalation Study of SCD-101 in Sickle Cell Disease

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

Allogeneic Stem Cell Transplantation Following Chemotherapy in Patients With Hemoglobinopathies