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Conditions: Infant, Newborn, Respiratory Insufficiency, Pulmonary Hypertension, Respiratory Distress Syndrome, Newborn, Streptococcal Infections
Interventions: Inhaled Prostaglandin E1; Drug
Lead sponsor: NICHD Neonatal Research Network; Network
Eligibility: Up to 7 Days
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008
U.S. locations: 11
States / cities: Birmingham, Alabama • Palo Alto, California • Iowa City, Iowa + 7 more

Conditions: Cancer
Interventions: itraconazole, voriconazole; Drug
Lead sponsor: Jonsson Comprehensive Cancer Center; Other
Eligibility: 12 Years to 120 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2003
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Prematurity, Bronchopulmonary Dysplasia, Intraventricular Hemorrhage, Periventricular Leukomalacia
Interventions: inhaled nitric oxide, oxygen; Drug
Lead sponsor: University of Chicago; Other
Eligibility: 2 Hours to 72 Hours
Enrollment: 273 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2023
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Anemia, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Diseases
Interventions: filgrastim, busulfan, fludarabine phosphate, methotrexate, tacrolimus, allogeneic bone marrow transplantation, allogeneic hematopoietic stem cell transplantation, peripheral blood stem cell transplantation; Biological · Drug · Procedure
Lead sponsor: University of California, San Francisco; Other
Eligibility: 16 Years to 60 Years
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2002
U.S. locations: 1
States / cities: San Francisco, California

Conditions: Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
Interventions: TAK-935, Placebo; Drug
Lead sponsor: Takeda; Industry
Eligibility: 2 Years to 17 Years
Enrollment: 141 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2020
U.S. locations: 15
States / cities: Phoenix, Arizona • Los Angeles, California • Aurora, Colorado + 12 more

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Nonmalignant Neoplasm
Interventions: trivalent influenza vaccine, Immunoenzyme technique, Laboratory biomarker analysis; Biological · Other
Lead sponsor: City of Hope Medical Center; Other
Eligibility: 18 Years to 120 Years
Enrollment: 459 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2007 – 2013
U.S. locations: 2
States / cities: Duarte, California • Pasadena, California

Conditions: Fabry Disease
Interventions: pegunigalsidase alfa; Drug
Lead sponsor: Chiesi Farmaceutici S.p.A.; Industry
Eligibility: 18 Years and older
Enrollment: 29 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2026
U.S. locations: 7
States / cities: Birmingham, Alabama • Atlanta, Georgia • Iowa City, Iowa + 4 more

Conditions: Brain and Central Nervous System Tumors, Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Unspecified Adult Solid Tumor, Protocol Specific
Interventions: educational intervention; Other
Lead sponsor: Case Comprehensive Cancer Center; Other
Eligibility: 18 Years to 120 Years
Enrollment: 583 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2012
U.S. locations: 4
States / cities: Detroit, Michigan • Cleveland, Ohio • Philadelphia, Pennsylvania

Conditions: Bronchopulmonary Dysplasia (BPD), Hypertension, Pulmonary
Interventions: Not listed
Lead sponsor: Stanford University; Other
Eligibility: Up to 30 Weeks
Enrollment: 40 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2019
U.S. locations: 2
States / cities: Mountain View, California • Palo Alto, California

Conditions: Fabry Disease
Interventions: DRX005B; Drug
Lead sponsor: National Institute of Neurological Disorders and Stroke (NINDS); NIH
Eligibility: Not listed
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2002 – 2003
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Leukemia, Lymphoma, Multiple Myeloma, Plasma Cell Neoplasm, Myelodysplastic Syndromes
Interventions: human myeloid progenitor cells; Biological
Lead sponsor: Cellerant Therapeutics; Industry
Eligibility: 12 Years to 65 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2014
U.S. locations: 8
States / cities: Orange, California • Wilmington, Delaware • Washington D.C., District of Columbia + 5 more

Conditions: Heart Defects, Congenital
Interventions: Clopidogrel, placebo; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 12 Months and older
Enrollment: 49 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2010
U.S. locations: 1
States / cities: Bridgewater, New Jersey

Conditions: Fabry Disease
Interventions: Venglustat (GZ402671), Placebo; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 16 Years and older
Enrollment: 122 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 12
States / cities: Birmingham, Alabama • Los Angeles, California • Orange, California + 9 more

Conditions: Anarthria, Dysarthria, Tetraplegia, Spinal Cord Injuries, Amyotrophic Lateral Sclerosis, Brain Stem Infarctions, Locked in Syndrome, Muscular Dystrophies
Interventions: BrainGate Neural Interface System; Device
Lead sponsor: Leigh R. Hochberg, MD, PhD.; Other
Eligibility: 18 Years to 80 Years
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2027
U.S. locations: 2
States / cities: Stanford, California • Boston, Massachusetts

Conditions: Advanced Solid Tumors
Interventions: Elimusertib (BAY1895344), Pembrolizumab (Keytruda®); Drug
Lead sponsor: Bayer; Industry
Eligibility: 18 Years and older
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2023
U.S. locations: 8
States / cities: Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more

Conditions: Heart Failure, Congenital Heart Disease
Interventions: rest-exercise echocardiography; Diagnostic Test
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years to 99 Years
Enrollment: 32 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2022
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic Syndromes, Myelodysplastic/Myeloproliferative Neoplasms, Precancerous Condition
Interventions: therapeutic allogeneic lymphocytes, busulfan, cyclosporine, fludarabine phosphate, mycophenolate mofetil, peripheral blood stem cell transplantation, Total Body Irradiation (TBI), Granulocyte colony-stimulating factor (G-CSF), Phenytoin, Methotrexate; Biological · Drug · Procedure + 1 more
Lead sponsor: OHSU Knight Cancer Institute; Other
Eligibility: 18 Years to 80 Years
Enrollment: 147 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2015
U.S. locations: 1
States / cities: Portland, Oregon

Conditions: Cavernous Angioma, Familial, Cerebral Cavernous Malformations, Cerebral Cavernous Hemangioma
Interventions: Simvastatin; Drug
Lead sponsor: University of New Mexico; Other
Eligibility: 18 Years and older
Enrollment: 12 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2016
U.S. locations: 1
States / cities: Albuquerque, New Mexico

Conditions: Autonomic Nervous System Diseases, Orthostatic Hypotension, Dopamine Beta-Hydroxylase Deficiency, Orthostatic Intolerance
Interventions: Droxidopa Oral Product; Drug
Lead sponsor: Vanderbilt University; Other
Eligibility: 18 Years to 70 Years
Enrollment: 8 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2020
U.S. locations: 1
States / cities: Nashville, Tennessee

Conditions: Non-infectious Uveitis
Interventions: AIN457, AIN 457; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 76 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 20
States / cities: Beverly Hills, California • Los Angeles, California • Sacramento, California + 16 more

Conditions: Cryopyrin-Associated Periodic Syndromes, Amyloidosis
Interventions: 124I AT-01; Drug
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 120 Years
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Pulmonary Hypertension
Interventions: Nitric Oxide/INP Pulse Delivery, Nitric Oxide/INO Pulse Delivery; Drug
Lead sponsor: National Heart, Lung, and Blood Institute (NHLBI); NIH
Eligibility: 18 Years and older
Enrollment: 31 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2006 – 2010
U.S. locations: 1
States / cities: Bethesda, Maryland

Conditions: Cystic Fibrosis
Interventions: Acute Antioxidant, Chronic Antioxidant, Placebo; Dietary Supplement · Other
Lead sponsor: Augusta University; Other
Eligibility: 7 Years and older
Enrollment: 13 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2015 – 2027
U.S. locations: 1
States / cities: Augusta, Georgia

Conditions: Mitochondrial Diseases, Maternally Inherited Diabetes and Deafness (MIDD), Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS), Mitochondrial DNA tRNALeu(UUR) m.3243A<G Mutation
Interventions: Sonlicromanol, Placebo; Drug
Lead sponsor: Khondrion BV; Industry
Eligibility: 18 Years and older
Enrollment: 220 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 3
States / cities: Boston, Massachusetts • Cleveland, Ohio • Houston, Texas

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Inhaled PGE1 in Neonatal Hypoxemic Respiratory Failure

Voriconazole Compared With Itraconazole in Preventing Fungal Infections in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Inhaled Nitric Oxide and Neuroprotection in Premature Infants

Busulfan and Fludarabine Before Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer

A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies

Flu Vaccine in Preventing Influenza Infection in Healthy Volunteers and in Patients Who Have Undergone Stem Cell Transplant

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Personalized Information or Basic Information in Helping Patients Make Decisions About Participating in a Clinical Trial

Predictors of Pulmonary Hypertension Risk in Premature Infants With Bronchopulmonary Dysplasia

Alpha-Galactosidase A Replacement Therapy for Fabry Disease

Safety Study of Human Myeloid Progenitor Cells (CLT-008) After Cord Blood Transplant for Hematologic Malignancy

Long Term Safety of Clopidogrel in Neonates/Infants With Systemic to Pulmonary Artery Shunt Palliation

A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

Feasibility of the BrainGate2 Neural Interface System in Persons With Tetraplegia

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

Ventricular and Pulmonary Vascular Reserve After the Fontan Operation

Busulfan, Fludarabine, and Total-Body Irradiation in Treating Patients Who Are Undergoing a Donor Stem Cell Transplant for Hematologic Cancer

Permeability MRI in Cerebral Cavernous Malformations Type 1 in New Mexico: Effects of Statins

Treatment of Orthostatic Hypotension

Safety and Efficacy of AIN457 in Noninfectious Uveitis

Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load

Cardiopulmonary Function Assessment and NO-Based Therapies for Patients With Hemolysis-Associated Pulmonary Hypertension

Mechanisms for Vascular Dysfunction and Exercise Tolerance in CF

KHENERFIN Study: A Trial to Evaluate the Efficacy and Safety of Sonlicromanol in Primary Mitochondrial Diseases