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Conditions: Priapism, Sickle Cell Disease, Hypogonadism
Interventions: Transdermal Androgel; Drug
Lead sponsor: Johns Hopkins University; Other
Eligibility: 18 Years to 50 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2016
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Anemia, Cooley's, Beta-Thalassemia, Hematologic Diseases, Thalassemia, Osteoporosis, Iron Overload, Hypertension, Pulmonary
Interventions: Deferoxamine, Deferiprone, Arginine, Sildenafil, Decitabine; Drug
Lead sponsor: Carelon Research; Other
Eligibility: 1 Year to 75 Years
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2000 – 2006
U.S. locations: 4
States / cities: Oakland, California • Boston, Massachusetts • New York, New York + 1 more

Conditions: Leukemia, Acute Lymphocytic (ALL), Leukemia, Myeloid, Acute(AML), Leukemia, Myeloid, Chronic(CML), Juvenile Myelomonocytic Leukemia(JMML), Hemoglobinuria, Paroxysmal Nocturnal (PNH), Lymphoma, Non-Hodgkin (NHL), Myelodysplastic Syndrome (MDS)
Interventions: Miltenyi Biotec CliniMACS, Stem Cell Transplantation, TBI, systemic chemotherapy and antibodies as follows:; Device · Procedure · Drug
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: 2 Years to 21 Years
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2009
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: Sickle Cell Disease
Interventions: SCD Shared Decision-Making Toolkit for AYAs - including VR, SCD Shared Decision-Making Toolkit for AYAs - without VR; Behavioral
Lead sponsor: Nemours Children's Clinic; Other
Eligibility: 15 Years and older
Enrollment: 68 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Wilmington, Delaware

Conditions: Sickle Cell Disease
Interventions: ATG, fludarabine, cyclophosphamide, Total body irradiation, Stem cell infusion, Sirolimus, mycophenolate mofetil; Drug · Radiation · Procedure
Lead sponsor: University of Illinois Chicago; Other
Eligibility: 16 Years to 60 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2029
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Sickle Cell Disease, Asthma
Interventions: Not listed
Lead sponsor: Nemours Children's Clinic; Other
Eligibility: 6 Years to 20 Years
Enrollment: 99 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 3
States / cities: Wilmington, Delaware • Jacksonville, Florida • Orlando, Florida

Conditions: Thalassemia, Iron Overload
Interventions: Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO); Drug
Lead sponsor: Elliott Vichinsky; Other
Eligibility: 8 Years and older
Enrollment: 22 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 1
States / cities: Oakland, California

Conditions: Thalassemia
Interventions: Busulfan, Fludarabine, Campath 1H, Cyclophosphamide, MESNA; Drug
Lead sponsor: Baylor College of Medicine; Other
Eligibility: Up to 64 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2016
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Sickle Cell Disease, Pain Management
Interventions: Hypnosis, Control; Other
Lead sponsor: National Institutes of Health Clinical Center (CC); NIH
Eligibility: 18 Years and older
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 2
States / cities: Washington D.C., District of Columbia • Bethesda, Maryland

Conditions: Thalassemia, Hypertension, Pulmonary
Interventions: Sildenafil; Drug
Lead sponsor: Carelon Research; Other
Eligibility: 7 Years and older
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2010
U.S. locations: 1
States / cities: Oakland, California

Conditions: Sickle Cell Disease, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
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 35 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2030
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Sickle Cell Disease
Interventions: Riociguat, Placebo; Drug
Lead sponsor: Mark Gladwin; Other
Eligibility: 18 Years and older
Enrollment: 130 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 20
States / cities: Oakland, California • Washington D.C., District of Columbia • Miami, Florida + 17 more

Conditions: Acne Vulgaris
Interventions: ACZONE Gel, 5%, Vehicle; Drug
Lead sponsor: Allergan; Industry
Eligibility: 12 Years and older
Enrollment: 64 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 29
States / cities: Birmingham, Alabama • San Diego, California • San Francisco, California + 24 more

Conditions: Sickle Cell Disease
Interventions: Crovalimab, Placebo; Drug
Lead sponsor: Hoffmann-La Roche; Industry
Eligibility: 12 Years to 55 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 2
States / cities: Atlanta, Georgia • New York, New York

Conditions: Acute Kidney Injury, Hemolysis, Thrombocytopenia, Surgery
Interventions: Non-pulsatile blood flow, Pulsatile blood flow; Other
Lead sponsor: University of Colorado, Denver; Other
Eligibility: 18 Years to 85 Years
Enrollment: 1,100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: Aurora, Colorado

Conditions: Pharyngitis, Infective
Interventions: Comparison between GenePOC CR and Reference Method; Device
Lead sponsor: Meridian Bioscience, Inc.; Industry
Eligibility: 25 Months and older
Enrollment: 497 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018
U.S. locations: 5
States / cities: Madera, California • Indianapolis, Indiana • Detroit, Michigan + 2 more

Conditions: AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions: Not listed
Lead sponsor: National Human Genome Research Institute (NHGRI); NIH
Eligibility: 1 Month to 100 Years
Enrollment: 999 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2099
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease
Interventions: Femoral Nerve Block; Procedure
Lead sponsor: Icahn School of Medicine at Mount Sinai; Other
Eligibility: 18 Years and older
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2013
U.S. locations: 1
States / cities: New York, New York

Conditions: Sickle Cell Disease
Interventions: Cardiac magnetic resonance imaging (CMR); Other
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 6 Years to 65 Years
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2019
U.S. locations: 1
States / cities: Cincinnati, Ohio

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 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: Paroxysmal Nocturnal Hemoglobinuria (PNH), Paroxysmal Hemoglobinuria
Interventions: Pegcetacoplan; Drug
Lead sponsor: Apellis Pharmaceuticals, Inc.; Industry
Eligibility: 12 Years to 17 Years
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Thalassemia
Interventions: Not listed
Lead sponsor: Carelon Research; Other
Eligibility: 12 Years to 90 Years
Enrollment: 252 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2010
U.S. locations: 7
States / cities: Oakland, California • Atlanta, Georgia • Chicago, Illinois + 4 more

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

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Androgen Regulation of Priapism in Sickle Cell Disease

Thalassemia (Cooley's Anemia) Clinical Research Network (TCRN)

Haploidentical Stem Cell Transplantation for Patients With Hematologic Malignancies

Empowering Adolescents and Young Adults With Sickle Cell Disease as Partners in Treatment Decision Making (EMPOWER-AYA)

Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease

Molecular Phenotyping of Asthma in Sickle Cell Disease

Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload

Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO

Hypnosis to Manage Pain and Symptoms in Patients With Sickle Cell Disease

Sildenafil to Improve Exercise Capacity in People With Thalassemia and Pulmonary Hypertension

Hematopoietic Stem Cell BCL11A Enhancer Gene Editing for Sickle Cell Disease

A Multi-Center Study of Riociguat in Patients With Sickle Cell Diseases

Phase IV Study to Gather More Information About the Safety of ACZONE Gel, 5% in Treating Subjects With Acne Who Have G6PD Deficiency

A Study Evaluating the Safety, Pharmacokinetics, Pharmacodynamics and Efficacy of Crovalimab for the Management of Acute Uncomplicated Vaso-Occlusive Episodes (VOE) in Participants With Sickle Cell Disease (SCD).

Optimizing Pulsatility During Cardiopulmonary Bypass to Reduce Acute Kidney Injury

Validation of the GenePOC Strep A, C/G Assay for Detecting Group A, C and G β-hemolytic Streptococcus From Throat Swabs.

A Natural History Study Seeks to Understand the Clinical, Genomic, Pharmacological, Laboratory, and Dietary Determinates of Pyrimidine and Purine Metabolism Disorders

Regional Anesthesia for Sickle Cell Crisis Using Ultrasound in The Emergency Department: Phase I

Novel Cardiac Magnetic Resonance Imaging to Define a Unique Restrictive Cardiomyopathy in Sickle Cell Disease

Bone Marrow for Hemoglobinopathy Research

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

A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Assessment of Pain in People With Thalassemia

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