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Conditions: Fetal Growth Restriction (FGR), Placental Insufficiency, Preeclampsia, Still Births, Pregnancy, Pregnancy Complications, Pregnancy Outcomes, Ultrasound
Interventions: ultrafast power Doppler imaging (uPDI) / quantitative ultrasound (QUS); Device
Lead sponsor: Carilion Clinic; Other
Eligibility: 18 Years to 45 Years · Female only
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Roanoke, Virginia

Conditions: Pregnancy Complications, Pregnancy Outcome, Congenital Abnormalities
Interventions: Observational Intervention; Biological
Lead sponsor: Emergent BioSolutions; Industry
Eligibility: Female only
Enrollment: 98 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2020
U.S. locations: 1
States / cities: San Diego, California

Conditions: Intraventricular Haemorrhage Neonatal, Death; Neonatal
Interventions: Umbilical cord milking UCM, Delayed cord clamping DCC; Procedure
Lead sponsor: Sharp HealthCare; Other
Eligibility: 23 Weeks to 33 Weeks
Enrollment: 1,201 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 15
States / cities: Birmingham, Alabama • Loma Linda, California • Los Angeles, California + 10 more

Conditions: Hunter Syndrome
Interventions: Idursulfase-IT, Elaprase; Drug
Lead sponsor: Takeda; Industry
Eligibility: 3 Years to 18 Years · Male only
Enrollment: 15 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2010 – 2024
U.S. locations: 7
States / cities: Chicago, Illinois • Chapel Hill, North Carolina • Portland, Oregon + 4 more

Conditions: Mucopolysaccharidosis (MPS)
Interventions: Not listed
Lead sponsor: Shire; Industry
Eligibility: Up to 7 Years · Male only
Enrollment: 159 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2015
U.S. locations: 7
States / cities: Los Angeles, California • Jacksonville, Florida • Minneapolis, Minnesota + 4 more

Conditions: MPS IIIB (Sanfilippo Syndrome)
Interventions: Not listed
Lead sponsor: Alexion Pharmaceuticals, Inc.; Industry
Eligibility: Not listed
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2015
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Mucopolysaccharidosis II
Interventions: Not listed
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: Male only
Enrollment: 36 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2022
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Colorectal Neoplasms
Interventions: Vicriviroc, Pembrolizumab; Drug · Biological
Lead sponsor: Merck Sharp & Dohme LLC; Industry
Eligibility: 18 Years and older
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 6
States / cities: Scottsdale, Arizona • Encinitas, California • Fresno, California + 3 more

Conditions: Tetralogy of Fallot, Ventricular Tachycardia, Sudden Cardiac Death
Interventions: Electroanatomic substrate mapping; Diagnostic Test
Lead sponsor: Boston Children's Hospital; Other
Eligibility: Not listed
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes, Inherited Metabolic Disorders (IMD), Hereditary Anemias, Inflammatory Conditions, Systemic Juvenile Idiopathic Arthritis (sJIA), Juvenile Rheumatoid Arthritis (JRA)
Interventions: Hydroxyurea, Alemtuzumab, Fludarabine, Melphalan, Thiotepa; Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 2 Months to 55 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Hunter Syndrome
Interventions: idursulfase-IT, No IT treatment; Biological · Other
Lead sponsor: Shire; Industry
Eligibility: Up to 18 Years · Male only
Enrollment: 58 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2017
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina

Conditions: Muscular Dystrophy, Arrhythmia, Sudden Cardiac Death
Interventions: Screening; Other
Lead sponsor: Indiana University; Other
Eligibility: 18 Years and older
Enrollment: 448 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1996 – 2015
U.S. locations: 1
States / cities: Indianapolis, Indiana

Conditions: Vitamin A Deficiency, Maternal Mortality, Infant Mortality
Interventions: Vitamin A or Beta-Carotene Supplements; Dietary Supplement
Lead sponsor: Johns Hopkins Bloomberg School of Public Health; Other
Eligibility: 15 Years to 49 Years · Female only
Enrollment: 59,666 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2001 – 2008
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: MPS II, Hunter Syndrome (MPS II)
Interventions: RGX-121-3102; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 4 Months to 5 Years · Male only
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2031
U.S. locations: 1
States / cities: New Brunswick, New Jersey

Conditions: Inherited Metabolic Diseases, Lysosomal Storage Disorders, Peroxisomal Storage Diseases, Inborn Errors of Metabolism, Mucopolysaccharidosis
Interventions: ALD-101; Biological
Lead sponsor: Aldagen; Industry
Eligibility: Up to 16 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2011
U.S. locations: 3
States / cities: Los Angeles, California • New York, New York • Durham, North Carolina

Conditions: Biliary Tract Cancer, Cholangiocarcinoma, Gallbladder Cancer
Interventions: M7824; Drug
Lead sponsor: EMD Serono Research & Development Institute, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 159 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2019 – 2022
U.S. locations: 7
States / cities: Scottsdale, Arizona • San Francisco, California • Jacksonville, Florida + 4 more

Conditions: Epilepsy, Seizures, Syncope, Channelopathy, Cardiomyopathies
Interventions: Not listed
Lead sponsor: Mayo Clinic; Other
Eligibility: 18 Years to 50 Years
Enrollment: 600 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2031
U.S. locations: 1
States / cities: Rochester, Minnesota

Conditions: Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Sepsis
Interventions: Glutamine, Placebo; Drug
Lead sponsor: NICHD Neonatal Research Network; Network
Eligibility: Up to 72 Hours
Enrollment: 1,433 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2001
U.S. locations: 15
States / cities: Birmingham, Alabama • Palo Alto, California • San Diego, California + 12 more

Conditions: Mucopolysaccharidosis Type I (MPS I), Mucopolysaccharidosis Type II (MPS II), Mucopolysaccharidosis Type III (MPS III), Mucopolysaccharidosis Type VI (MPS VI), Krabbe Disease
Interventions: Not listed
Lead sponsor: University of Chicago; Other
Eligibility: 1 Day to 18 Years
Enrollment: 19 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2016
U.S. locations: 3
States / cities: Chicago, Illinois • Minneapolis, Minnesota • Buffalo, New York

Conditions: Primary Immune Deficiency
Interventions: Not listed
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 1 Month to 100 Years
Enrollment: 5,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2007
U.S. locations: 2
States / cities: Bethesda, Maryland • Ann Arbor, Michigan

Conditions: Kidney Transplant
Interventions: Berinert, Placebo for Berinert; Biological · Other
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 75 Years
Enrollment: 180 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2032
U.S. locations: 4
States / cities: Los Angeles, California • Chicago, Illinois • Kansas City, Kansas + 1 more

Conditions: Mucopolysaccharidosis II
Interventions: Genetic HMI-203; Biological
Lead sponsor: Homology Medicines, Inc; Industry
Eligibility: 18 Years to 45 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2029
U.S. locations: 5
States / cities: Oakland, California • New Haven, Connecticut • Hackensack, New Jersey + 2 more

Conditions: Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC
Interventions: Not listed
Lead sponsor: Lexeo Therapeutics; Industry
Eligibility: 18 Years to 67 Years
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2030
U.S. locations: 1
States / cities: Ann Arbor, Michigan

Conditions: Hunter Syndrome
Interventions: Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg), Idursulfase IT (30 mg); Other · Drug
Lead sponsor: Shire; Industry
Eligibility: 3 Years to 18 Years · Male only
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2012
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

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Placental Imaging Techniques

BioThrax® (Anthrax) Vaccine in Pregnancy Registry

Premature Infants Receiving Milking or Delayed Cord Clamping: PREMOD2

An Extension Study of HGT-HIT-045 Evaluating Long-Term Safety and Clinical Outcomes of Idursulfase-IT in Conjunction With Elaprase in Pediatric Participants With Hunter Syndrome and Cognitive Impairment

Mucopolysaccharidosis (MPS) I, II, and VI Screening in a High-Risk Population With Previous Surgical Repair or Presence of Inguinal and/or Umbilical Hernia in Combination With Pediatric ENT Surgery (The HATT Project)

A Retrospective Chart Review of Deceased Patients With Mucopolysaccharidosis Type IIIB

Mucopolysaccharidosis Type II Natural History

Safety and Efficacy of Vicriviroc (MK-7690) in Combination With Pembrolizumab (MK-3475) in Participants With Advanced/Metastatic Microsatellite Stable (MSS) Colorectal Cancer (CRC) (MK-7690-046)

Electroanatomic Interactions Between Transcatheter Pulmonary Valve Prostheses and Anatomic Isthmuses in Repaired Tetralogy of Fallot

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment

Arrhythmias in Myotonic Muscular Dystrophy

Impact of Maternal Vitamin A or Beta-Carotene Supplementation on Maternal and Infant Mortality in Bangladesh

RGX-121-3102 Gene Therapy in Participants With MPS II (Hunter Syndrome)

ALD-101 Adjuvant Therapy of Unrelated Umbilical Cord Blood Transfusion (UCBT) in Patients With Inherited Metabolic Diseases

M7824 Monotherapy in Locally Advanced or Metastatic Second Line (2L) Biliary Tract Cancer (Cholangiocarcinoma and Gallbladder Cancer)

Genetic Markers of Cardiovascular Disease in Epilepsy

Glutamine Supplementation to Prevent Death or Infection in Extremely Premature Infants

Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children

Screening Protocol for Genetic Diseases of Lymphocyte Homeostasis and Programmed Cell Death

Improving Deceased-Donor Kidney Transplant Outcomes Via a Single Intragraft Injection of C1 Esterase Inhibitor (IMPROVE TRIAL)

Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II

Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®