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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: Sickle Cell Disease
Interventions: AB1; Drug
Lead sponsor: Nirmish Shah; Other
Eligibility: 18 Years to 45 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 3
States / cities: Augusta, Georgia • Durham, North Carolina • Greenville, North Carolina

Conditions: Pain, Sickle Cell Disorder
Interventions: Normal saline, Ketamine; Drug
Lead sponsor: Billy Sin; Other
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2018
U.S. locations: 1
States / cities: Brooklyn, New York

Conditions: Sickle Cell Disease
Interventions: Voxelotor; Drug
Lead sponsor: Pfizer; Industry
Eligibility: 6 Months to 17 Years
Enrollment: 147 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2023
U.S. locations: 13
States / cities: Brentwood, California • Washington D.C., District of Columbia • Atlanta, Georgia + 9 more

Conditions: Severe Aplastic Anemia (SAA), Hypo-Plastic Myelodysplastic Syndrome (MDS), Paroxysmal Nocturnal Hemoglobinuria (PNH)
Interventions: Cyclophosphamide, Peripheral Blood Stem Cells; Drug · Other
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 4 Years to 75 Years
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2028
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Chuvash Polycythemia, Cerebrovascular Disease, Pulmonary Hypertension, Sickle Cell Anemia
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 3 Years to 20 Years
Enrollment: 29 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2007 – 2014
U.S. locations: 6
States / cities: Washington D.C., District of Columbia • Chicago, Illinois • Bethesda, Maryland + 2 more

Conditions: Paroxysmal Nocturnal Hemoglobinuria
Interventions: Danicopan; Drug
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: Not listed
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2026
U.S. locations: 7
States / cities: Los Angeles, California • Chicago, Illinois • Baltimore, Maryland + 4 more

Conditions: Sickle Cell Disease
Interventions: Music therapy, Health education; Behavioral · Other
Lead sponsor: University of California, Irvine; Other
Eligibility: 14 Years and older
Enrollment: 90 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 2
States / cities: Cleveland, Ohio • Greenville, South Carolina

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: Hemoglobin Mutations, Erythrocyte Variants
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 65 Years
Enrollment: 130 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2006 – 2010
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Paroxysmal Nocturnal Hemoglobinuria
Interventions: Pegcetacoplan, Soliris; Drug
Lead sponsor: Apellis Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 19
States / cities: Los Angeles, California • Santa Monica, California • Denver, Colorado + 16 more

Conditions: Sickle Cell Disease
Interventions: ICL670; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years and older
Enrollment: 185 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2004
U.S. locations: 31
States / cities: Mobile, Alabama • Loma Linda, California • Los Angeles, California + 25 more

Conditions: Sickle Cell Anaemia, Sickle Cell Anemia Crisis, Sickle Cell Anemia in Children, Sickle Cell Anemia (HbSS, or HbSβ-thalassemia0)
Interventions: Hemanext ONE System, Conventional RBCs; Device
Lead sponsor: Hemanext; Industry
Eligibility: 7 Years and older
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2029
U.S. locations: 5
States / cities: Farmington, Connecticut • Atlanta, Georgia • Chicago, Illinois + 2 more

Conditions: Sickle Cell Disease
Interventions: Single Session Problem-Solving Intervention; Behavioral
Lead sponsor: University of Texas Southwestern Medical Center; Other
Eligibility: 7 Years and older
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2022
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Sickle Cell Disease
Interventions: voxelotor, Placebo; Drug · Other
Lead sponsor: Pfizer; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 449 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2019
U.S. locations: 29
States / cities: Birmingham, Alabama • Mobile, Alabama • Little Rock, Arkansas + 25 more

Conditions: Sickle Cell Disease, Vaso-Occlusive Pain Episode in Sickle Cell Disease, Ketamine Infusion, Pain Management, Vaso-Occlusive Crises
Interventions: Sub-dissociative Ketamine Infusion, Normal Saline Placebo Infusion; Drug
Lead sponsor: Mohsen Saidinejad; Other
Eligibility: 5 Years to 20 Years
Enrollment: 120 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Torrance, California

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: Atypical Hemolytic-Uremic Syndrome
Interventions: Not listed
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: Not listed
Enrollment: 3,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2031
U.S. locations: 9
States / cities: Aurora, Colorado • Washington D.C., District of Columbia • Gainesville, Florida + 6 more

Conditions: Sickle Cell Anemia, Sickle Cell Disease, Hemoglobin Disorder
Interventions: Metformin, Questionnaires; Drug · Behavioral
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 10 Years to 60 Years
Enrollment: 37 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2020
U.S. locations: 2
States / cities: Houston, Texas

Conditions: Autoimmune Hemolytic Anemia, Immune Thrombocytopenia
Interventions: KZR-616; Drug
Lead sponsor: Kezar Life Sciences, Inc.; Industry
Eligibility: 18 Years and older
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020
U.S. locations: 15
States / cities: Los Angeles, California • San Francisco, California • Jacksonville, Florida + 12 more

Conditions: Sickle Cell Anemia, Sickle Cell Thalassemia, Sickle Cell Pain, Hbss, Hbsc, Sickle Beta Thalassemia, Sickle Beta Zero Thalassemia, Sickle Cell Syndrome Variant
Interventions: Not listed
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 16 Years to 80 Years
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Sickle Cell Disease
Interventions: Plerixafor; Drug
Lead sponsor: City of Hope Medical Center; Other
Eligibility: 18 Years to 40 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2027
U.S. locations: 1
States / cities: Duarte, California

Conditions: Sickle Cell Disease
Interventions: Peer Mentoring [PM], Structured Education Based Transition Program STE; Other
Lead sponsor: Wake Forest University Health Sciences; Other
Eligibility: 16 Years and older
Enrollment: 291 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 14
States / cities: Birmingham, Alabama • Mobile, Alabama • Miami, Florida + 10 more

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 21 Years
Enrollment: 68 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2025
U.S. locations: 1
States / cities: Chicago, Illinois

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Fresh Versus Old Red Blood Cells for Transfusion

AB1 in Adult Patients With Sickle Cell Disease (SCD)

Ketamine Infusion for Acute Sickle Cell Crisis in the Emergency Department

Study to Evaluate the Effect of GBT440 in Pediatrics With Sickle Cell Disease

Haplo-identical Transplantation for Severe Aplastic Anemia, Hypo-plastic MDS and PNH Using Peripheral Blood Stem Cells and Post-transplant Cyclophosphamide for GVHD Prophylaxis

Pulmonary Hypertension, Hypoxia and Sickle Cell Disease

A Long-term Safety and Efficacy Study of Danicopan as an Add-on Therapy to Complement Component 5 Inhibitor (C5i) in Participants With PNH

MULTIsite Feasibility of MUSIc Therapy to Address Quality Of Life in Sickle Cell Disease

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

Collection of Blood From Persons With Hemoglobin and Erythrocyte Polymorphisms for Laboratory Malaria Research

Study to Evaluate the Efficacy and Safety of APL-2 in Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)

Evaluation of the Safety, Tolerability, Pharmacokinetics (PK) and Effects on Liver Iron Concentration of ICL670 Relative to Deferoxamine(DFO).

Hypoxic Red Blood Cells in Sickle Cell Anemia

Utilizing A Single Session Problem-Solving Intervention With Caregivers of Pediatric Patients Receiving Chronic Transfusion to Treat Sickle Cell Disease

Study to Evaluate the Effect of Voxelotor Administered Orally to Patients With Sickle Cell Disease (GBT_HOPE)

Sub-dissociative Dose Ketamine in Treatment of Vaso-occlusive Pain Event in Children and Young Adults

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Atypical Hemolytic-Uremic Syndrome (aHUS) Registry

Fetal Hemoglobin Induction Treatment Metformin

A Phase 2 Study to Evaluate the Safety and Efficacy of KZR-616 in Patients With AIHA and ITP

Investigating the Mechanistic Effects of Mitapivat in Subjects With Sickle Cell Disease

Safety of Blood Stem Cell Mobilization With Plerixafor in Patients With Sickle Cell Disease

LCI-HEM-SCD-ST3P-UP-001: The Sickle Cell Trevor Thompson Transition Project (ST3P-UP Study)

The Longitudinal Relationship of HU Adherence to HRQOL, Barriers to Adherence and Habit in SCD.