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Conditions: Sickle Cell Disease, Iron Overload, Hemolytic Anemia
Interventions: Deferasirox (ICL670), Deferoxamine (DFO); Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years and older
Enrollment: 212 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2008
U.S. locations: 56
States / cities: Birmingham, Alabama • Mobile, Alabama • Loma Linda, California + 32 more

Conditions: Thrombosis
Interventions: Not listed
Lead sponsor: Albert Einstein College of Medicine; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 34 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2010 – 2011
U.S. locations: 1
States / cities: The Bronx, New York

Conditions: Sickle Cell Disease
Interventions: GBT440; Drug
Lead sponsor: Global Blood Therapeutics; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 24 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2016
U.S. locations: 1
States / cities: San Antonio, Texas

Conditions: Sickle Cell Disease (SCD)
Interventions: Crizanlizumab (SEG101), Placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 12 Years to 100 Years
Enrollment: 253 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2026
U.S. locations: 5
States / cities: Atlanta, Georgia • Boston, Massachusetts • Charlotte, North Carolina + 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: Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
Interventions: Allogeneic stem cell transplant; Device
Lead sponsor: St. Jude Children's Research Hospital; Other
Eligibility: Up to 21 Years
Enrollment: 4 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2009
U.S. locations: 1
States / cities: Memphis, Tennessee

Conditions: G6PD Deficiency
Interventions: SD Biosensor STANDARD G6PD Test, Pointe Scientific Test Kit, HemoCue System; Diagnostic Test
Lead sponsor: PATH; Other
Eligibility: 18 Years to 65 Years
Enrollment: 229 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2020
U.S. locations: 1
States / cities: Reading, Pennsylvania

Conditions: Sickle Cell Disease, Vaso-occlusive Crisis, Acute Chest Syndrome
Interventions: A-001, Placebo; Drug · Other
Lead sponsor: Anthera Pharmaceuticals; Industry
Eligibility: 5 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 6
States / cities: Washington D.C., District of Columbia • Atlanta, Georgia • Chicago, Illinois + 3 more

Conditions: Sickle Cell Disease
Interventions: Cogmed+; Behavioral
Lead sponsor: Children's National Research Institute; Other
Eligibility: 7 Years to 16 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Hemoglobin SC Disease, Anemia, Sickle Cell
Interventions: Parent Educational Support; Behavioral
Lead sponsor: University of Miami; Other
Eligibility: 6 Years to 12 Years
Enrollment: 73 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2016
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Sickle Cell Disease, Vaso-occlusive Crisis
Interventions: Steroid arm, Placebo; Drug · Other
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 8 Years and older
Enrollment: 18 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2008
U.S. locations: 1
States / cities: Houston, Texas

Conditions: Sickle Cell Disease
Interventions: Liver transient elastography (Fibroscan), Ferriscan, Liver biopsy in iron overloaded subjects.; Other · Procedure
Lead sponsor: University of Miami; Other
Eligibility: 10 Years to 65 Years
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2017
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Anemia, Sickle Cell
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: Not listed
Enrollment: 3,640 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 13
States / cities: Oakland, California • San Francisco, California • Boston, Massachusetts + 6 more

Conditions: Sickle Cell Disease, Sickle Cell Anemia, Sickle Cell Disorders, Hemoglobin S Disease, Sickling Disorder Due to Hemoglobin S
Interventions: HQK-1001; Drug
Lead sponsor: HemaQuest Pharmaceuticals Inc.; Industry
Eligibility: 12 Years to 60 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2011
U.S. locations: 9
States / cities: Oakland, California • Miami, Florida • Augusta, Georgia + 6 more

Conditions: Fanconi Anemia, Unspecified Adult Solid Tumor, Protocol Specific, Unspecified Childhood Solid Tumor, Protocol Specific
Interventions: biologic sample preservation procedure, biopsy; Other · Procedure
Lead sponsor: OHSU Knight Cancer Institute; Other
Eligibility: 1 Year to 55 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2013
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Sickle Cell Disease (SCD)
Interventions: crizanlizumab; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 16 Years to 70 Years
Enrollment: 57 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 12
States / cities: Orange, Florida • Tampa, Florida • Atlanta, Georgia + 9 more

Conditions: Sickle Cell Disease, Sickle Cell Nephropathy
Interventions: Atorvastatin, Placebo; Drug
Lead sponsor: University of North Carolina, Chapel Hill; Other
Eligibility: 18 Years to 60 Years
Enrollment: 13 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2018
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: Sickle Cell Disease, Obesity, Childhood, Hypertension, Hyperlipidemia in Children, Hyperglycemia
Interventions: Not listed
Lead sponsor: University of Mississippi Medical Center; Other
Eligibility: 10 Years to 19 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Jackson, Mississippi

Conditions: Anemia, Sickle Cell, Pneumonia
Interventions: Arginine hydrochloride; Drug
Lead sponsor: UCSF Benioff Children's Hospital Oakland; Other
Eligibility: 5 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2005
U.S. locations: 1
States / cities: Oakland, California

Conditions: Sickle Cell Disease
Interventions: Voxelotor; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 6 Months to 11 Years
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 33
States / cities: Phoenix, Arizona • San Diego, California • Wilmington, Delaware + 20 more

Conditions: Anemia, Sickle Cell
Interventions: Nitric Oxide, Placebo; Drug
Lead sponsor: Mallinckrodt; Industry
Eligibility: 10 Years and older
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2008
U.S. locations: 11
States / cities: Birmingham, Alabama • Oakland, California • Aurora, Colorado + 7 more

Conditions: Sickle Cell Disease, Sickle Cell Anemia in Children, Sickle Cell Disease (SCD), Sickle Cell, Sickle Cell Anemia (HbSS)
Interventions: Individuation and Perspective-Taking (IPT), pediatric SCD pain management education session; Behavioral · Other
Lead sponsor: Connecticut Children's Medical Center; Other
Eligibility: 18 Years and older
Enrollment: 30 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2027 – 2030
U.S. locations: 1
States / cities: Hartford, Connecticut

Conditions: Sickle Cell Disease, Kidney Injury, Kidney Diseases, Kidney Disease, Chronic
Interventions: Not listed
Lead sponsor: University of Alabama at Birmingham; Other
Eligibility: 1 Year to 25 Years
Enrollment: 60 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017 – 2022
U.S. locations: 1
States / cities: Birmingham, Alabama

Conditions: Sickle Cell Disorders, Sickle Cell Disease, Hemoglobin S, Hb-SS Disease, Disease Sickle Cell Anemia
Interventions: Fostamatinib; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years to 65 Years
Enrollment: 35 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

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Study of Deferasirox Relative to Subcutaneous Deferoxamine in Sickle Cell Disease Patients

Sickle Cell Trait and the Risk of Venous Thromboembolism

Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects

Study of Two Doses of Crizanlizumab Versus Placebo in Adolescent and Adult Sickle Cell Disease Patients

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

Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias

Validation of a Diagnostic Test for Glucose-6-phosphate Dehydrogenase (G6PD) Deficiency in Anti-coagulated Blood

IMPACTS Trial: Investigation of the Modulation of Phospholipase in Acute Chest Syndrome

Advancing Feasibility and Acceptability of Digital Cognitive Rehabilitation in Sickle Cell Disease

Parent Educational Program for Children With Sickle Cell Disease

Steroid Treatment for Sickle Cell Pain Crisis

Liver Fibrosis in Sickle Cell Disease

Establishing a Repository of Blood and DNA Samples From People With Sickle Cell Disease (Comprehensive Sickle Cell Centers Collaborative Genotype-Phenotype Database and Sample Repository)

A Study of HQK-1001 in Patients With Sickle Cell Disease

Tissue Sample Collection From Patients With Fanconi Anemia

Pharmacokinetics and Pharmacodynamics Study of SEG101 (Crizanlizumab) in Sickle Cell Disease (SCD) Patients With Vaso- Occlusive Crisis (VOC)

Effect of Atorvastatin on Endothelial Dysfunction and Albuminuria in Sickle Cell Disease

Obesity in Pediatric Sickle Cell Disease: A New Phenomenon

Arginine Treatment of Acute Chest Syndrome (Pneumonia) in Sickle Cell Disease Patients

Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options

Nitric Oxide Inhalation to Treat Sickle Cell Pain Crises

Development of a Provider-Focused Intervention to Improve Health Outcomes in Pediatric Sickle Cell Disease

Acute Kidney Injury in Patients With Sickle Cell Disease

Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease