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Conditions: Vitamin D Deficiency, Stem Cell Transplant Complications, Pediatric Cancer, Blood Disorder, Pediatric Acute Myeloid Leukemia, Pediatric Acute Lymphoid Leukemia, Myelodysplastic Syndromes, Sickle Cell Anemia in Children, Aplastic Anemia, Thalassemia in Children
Interventions: Vitamin D3, Standard Vitamin D3 Supplementation; Dietary Supplement
Lead sponsor: Phoenix Children's Hospital; Other
Eligibility: 1 Year to 25 Years
Enrollment: 49 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: Phoenix, Arizona

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: Thalassemia
Interventions: Luspatercept, Placebo, Best Supportive Care (BSC); Drug · Other
Lead sponsor: Celgene; Industry
Eligibility: 18 Years and older
Enrollment: 145 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 4
States / cities: Los Angeles, California • Oakland, California • Chicago, Illinois

Conditions: Healthy Volunteers Sickle Cell Disease, Thalassemia
Interventions: Etavopivat, Digoxin, Pitavastatin, Metformin, Midazolam, Rosuvastatin; Drug
Lead sponsor: Novo Nordisk A/S; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 37 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025
U.S. locations: 1
States / cities: Salt Lake City, Utah

Conditions: Beta-Thalassemia
Interventions: ICL670, deferoxamine; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years and older
Enrollment: 595 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2003
U.S. locations: 7
States / cities: Los Angeles, California • Oakland, California • Stanford, California + 4 more

Conditions: Transfusional Iron Overload, Beta-thalassemia
Interventions: FBS0701; Drug
Lead sponsor: FerroKin BioSciences, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 20 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2009
U.S. locations: 3
States / cities: Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania

Conditions: Thalassemia
Interventions: Decitabine (USAN, INN); Drug
Lead sponsor: Carelon Research; Other
Eligibility: 18 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 2
States / cities: Oakland, California • Philadelphia, Pennsylvania

Conditions: Non-Transfusion Dependent Beta-Thalassemia (NTDT)
Interventions: REGN7999, Placebo; Drug
Lead sponsor: Regeneron Pharmaceuticals; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: New York, New York

Conditions: Sickle Cell Anemia, Thalassemia
Interventions: L-glutamine, Placebo; Drug
Lead sponsor: Emmaus Medical, Inc.; Industry
Eligibility: 5 Years and older
Enrollment: 70 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2008
U.S. locations: 5
States / cities: Bellflower, California • Torrance, California • Atlanta, Georgia + 2 more

Conditions: Thalassemia Major
Interventions: Vitamin D3, Placebo; Drug
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 6 Years to 60 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2014
U.S. locations: 1
States / cities: New York, New York

Conditions: Thalassemia, Sickle Cell Disease, Diamond Blackfan Anemia, Myelofibrosis
Interventions: Deferasirox; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years and older
Enrollment: 1,683 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2008
U.S. locations: 27
States / cities: Little Rock, Arkansas • Berkeley, California • Orange, California + 23 more

Conditions: Transfusion-dependent Alpha-Thalassemia, Transfusion-dependent Beta-Thalassemia
Interventions: Placebo Matching Mitapivat, Mitapivat; Drug
Lead sponsor: Agios Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 258 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2029
U.S. locations: 10
States / cities: Phoenix, Arizona • La Jolla, California • Oakland, California + 7 more

Conditions: Sickle Cell Disease
Interventions: IMR-687, Placebo; Drug
Lead sponsor: Cardurion Pharmaceuticals, Inc.; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 7
States / cities: Oakland, California • Farmington, Connecticut • Hollywood, Florida + 3 more

Conditions: Erythrocyte Transfusion, Beta-Thalassemia
Interventions: Luspatercept, Placebo; Drug · Other
Lead sponsor: Celgene; Industry
Eligibility: 18 Years and older
Enrollment: 336 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2021
U.S. locations: 6
States / cities: Los Angeles, California • Oakland, California • Chicago, Illinois + 3 more

Conditions: Transfusional Iron Overload
Interventions: ICL670; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 2 Years and older
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2003
U.S. locations: 8
States / cities: Oakland, California • Orange, California • Boston, Massachusetts + 4 more

Conditions: High Risk Pregnancy, Congenital Heart Disease, Fetal Hydrops, Twin Monochorionic Monoamniotic Placenta, Gastroschisis, Fetal Demise, Stillbirth, Fetal Arrhythmia, Long QT Syndrome, Intrauterine Fetal Death, Sudden Infant Death, Pregnancy Loss, Twin Twin Transfusion Syndrome, Birth Defect, Fetal Cardiac Anomaly, Fetal Cardiac Disorder, Fetal Death, Brugada Syndrome, Fetal Tachycardia
Interventions: Fetal Magnetocardiogram and Neonatal Electrocardiogram, Substudy only: Maternal/Infant Pharmacogenomic assessment postnatally; Diagnostic Test · Genetic
Lead sponsor: Medical College of Wisconsin; Other
Eligibility: 18 Years and older · Female only
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2028
U.S. locations: 2
States / cities: Madison, Wisconsin • Milwaukee, Wisconsin

Conditions: Sickle Cell Disease, HEMOGLOBIN SS, Hemoglobin S Beta-0 Thalassemia, Inflammation, Quality of Life
Interventions: Omega-3 Fatty Acids: Eicosapentaenoic Acid (EPA) and Docosahexaenoic Acid (DHA), Placebo Capsules; Dietary Supplement · Other
Lead sponsor: Thomas Jefferson University; Other
Eligibility: 10 Years to 19 Years
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2012
U.S. locations: 2
States / cities: Philadelphia, Pennsylvania

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: Thalassemia
Interventions: Not listed
Lead sponsor: Carelon Research; Other
Eligibility: 5 Years and older
Enrollment: 416 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2007 – 2011
U.S. locations: 10
States / cities: Los Angeles, California • Oakland, California • Stanford, California + 7 more

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: Beta-Thalassemia
Interventions: No Intervention; Other
Lead sponsor: Genetix Biotherapeutics Inc.; Industry
Eligibility: Not listed
Enrollment: 150 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2043
U.S. locations: 6
States / cities: Oakland, California • Palo Alto, California • Minneapolis, Minnesota + 3 more

Conditions: Thalassemia, Iron Overload, Hemochromatosis
Interventions: Nifedipine, Phlebotomy, Urinalysis, Echocardiogram, Electrocardiogram; Drug · Procedure
Lead sponsor: National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); NIH
Eligibility: 18 Years and older
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Hydrops Fetalis, Fetal Growth Retardation
Interventions: Tandem MS test for inborn errors of metabolism; Procedure
Lead sponsor: Obstetrix Medical Group; Industry
Eligibility: 18 Years to 60 Years · Female only
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2009
U.S. locations: 11
States / cities: Phoenix, Arizona • Laguna Hills, California • Long Beach, California + 6 more

Conditions: β-thalassemia, Ineffective Erythropoiesis, Chronic Anemia
Interventions: PTG-300; Drug
Lead sponsor: Protagonist Therapeutics, Inc.; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 63 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 3
States / cities: Oakland, California • Boston, Massachusetts • Philadelphia, Pennsylvania

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A Randomized Phase IV Control Trial of Single High Dose Oral Vitamin D3 in Pediatric Patients Undergoing HSCT

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

A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia

A Research Study of the Effect of Etavopivat on Other Drugs in Healthy Participants

Safety & Efficacy of ICL670 vs. Deferoxamine in Beta-thalassemia Patients With Iron Overload Due to Blood Transfusions

Safety and Pharmacokinetic Study of Escalating Multiple Doses of an Iron Chelator in Patients With Iron Overload

Evaluating the Safety and Effectiveness of Decitabine in People With Thalassemia Intermedia

A Study to Test the Safety, Tolerability, and Efficacy of an Antibody, REGN7999, Injected Under the Skin for the Treatment of Iron Overload in Adult Participants With Non-Transfusion Dependent β-thalassemia, Using MRI Scans to Measure Iron Levels in the Body

L-Glutamine Therapy for Sickle Cell Anemia and Sickle ß0 Thalassemia

The Effect of Vitamin D Supplementation on Calcium Excretion in Thalassemia: a Dose Response Study

Expanded Access of Deferasirox to Patients With Congenital Disorders of Red Blood Cells and Chronic Iron Overload

A Study Evaluating the Efficacy and Safety of Mitapivat in Participants With Transfusion-Dependent Alpha- or Beta-Thalassemia (α- or β-TDT)

A Study of IMR-687 in Adult Participants With Sickle Cell Anemia (Homozygous HbSS or Sickle-β0 Thalassemia)

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

A Protocol to Allow Treatment With ICL670 for Patients With or at Risk of Life-threatening Complications of Transfusional Iron Overload Who Are Unable to Tolerate Other Iron Chelators Because of Documented Severe Toxicity

Fetal Electrophysiologic Abnormalities in High-Risk Pregnancies Associated With Fetal Demise

A Randomized Trial of LOVAZA in Pediatric Sickle Cell Disease (SCD)

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

Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study

Efficacy of Vorinostat to Induce Fetal Hemoglobin in Sickle Cell Disease

A Study of Participants with Β-Thalassemia Treated with Betibeglogene Autotemcel

Oral Nifedipine to Treat Iron Overload

Amniotic Fluid Tandem Mass Spectrometry for Pregnancies Complicated by NIH and Severe Symmetrical IUGR

Study of PTG-300 in Non-Transfusion Dependent and Transfusion-Dependent Beta-Thalassemia Subjects With Chronic Anemia