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Conditions: Environmental Tobacco Smoke Exposure, Depression, Partner Abuse, Tobacco Smoking
Interventions: Cognitive Behavioral Therapy; Behavioral
Lead sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD); NIH
Eligibility: 18 Years and older · Female only
Enrollment: 1,750 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2001 – 2004
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Mucopolysaccharidosis Type II (MPS II)
Interventions: RGX-121; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 4 Months to 5 Years · Male only
Enrollment: 48 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2025
U.S. locations: 4
States / cities: Oakland, California • New Brunswick, New Jersey • Philadelphia, Pennsylvania + 1 more

Conditions: Perinatal Death, Respiratory Distress Syndrome, Newborn, Hypoxic-Ischemic Encephalopathy, Neonatal Seizure, Meconium Aspiration Syndrome, Intracranial Hemorrhages, Neonatal Hypotension
Interventions: Room air; Other
Lead sponsor: Loma Linda University; Other
Eligibility: 18 Years to 55 Years · Female only
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2022
U.S. locations: 1
States / cities: Loma Linda, California

Conditions: Infant Death, Sudden Infant Death
Interventions: TodaysBaby; Behavioral
Lead sponsor: University of Virginia; Other
Eligibility: Female only
Enrollment: 10,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2028
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Infant, Newborn, Infant, Low Birth Weight, Infant, Small for Gestational Age, Infant, Premature, Cerebral Palsy
Interventions: Not listed
Lead sponsor: NICHD Neonatal Research Network; Network
Eligibility: Up to 72 Hours
Enrollment: 1,067 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 1999 – 2004
U.S. locations: 18
States / cities: Birmingham, Alabama • Palo Alto, California • San Diego, California + 14 more

Conditions: Sudden Infant Death
Interventions: Safety Baby shower, Standard of Care; Behavioral
Lead sponsor: Arkansas Children's Hospital Research Institute; Other
Eligibility: 13 Years to 19 Years · Female only
Enrollment: 146 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2020
U.S. locations: 1
States / cities: Little Rock, Arkansas

Conditions: Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III), Krabbe Disease (Globoid Leukodystrophy), Metachromatic Leukodystrophy (MLD), Adrenoleukodystrophy (ALD and AMN), Sandhoff Disease, Tay Sachs Disease, Pelizaeus Merzbacher (PMD), Niemann-Pick Disease, Alpha-mannosidosis
Interventions: hematopoietic stem cell infusion; Biological
Lead sponsor: Talaris Therapeutics Inc.; Industry
Eligibility: Not listed
Enrollment: 3 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2011 – 2016
U.S. locations: 1
States / cities: Durham, North Carolina

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: MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease
Interventions: Aldurazyme (laronidase); Drug
Lead sponsor: University of California, San Francisco; Other
Eligibility: 18 Years to 50 Years · Female only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2032
U.S. locations: 1
States / cities: San Francisco, California

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: 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: Mucopolysaccharidosis II, MPS II
Interventions: SB-913; Biological
Lead sponsor: Sangamo Therapeutics; Industry
Eligibility: 5 Years to 65 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2021
U.S. locations: 5
States / cities: Oakland, California • Chicago, Illinois • New York, New York + 2 more

Conditions: Sudden Infant Death, Sudden Unexplained Infant Death
Interventions: ISA-MI, Standard of Care (SOC); Behavioral · Other
Lead sponsor: Johns Hopkins University; Other
Eligibility: 3 Days to 75 Years
Enrollment: 350 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2022 – 2027
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Hunter Syndrome
Interventions: Idursulfase; Biological
Lead sponsor: Shire; Industry
Eligibility: 5 Years and older · Male only
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 2
States / cities: Oakland, California • Minneapolis, Minnesota

Conditions: Hunter Syndrome, Mucopolysaccharidosis II (MPS II)
Interventions: Idursulfase; Biological
Lead sponsor: Shire; Industry
Eligibility: 5 Years and older · Male only
Enrollment: 94 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2008
U.S. locations: 16
States / cities: Phoenix, Arizona • Oakland, California • Denver, Colorado + 13 more

Conditions: Infant Death, Serious Injury
Interventions: SNOO Bassinet; Device
Lead sponsor: Happiest Baby, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 1,900 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2028
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
Interventions: Somatropin (DNA origin); Drug
Lead sponsor: Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center; Other
Eligibility: 5 Years to 17 Years
Enrollment: 2 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 1
States / cities: Minneapolis, Minnesota

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: MLD, Krabbe Disease, ALD, MPS I, MPS II, MPS III, Vanishing White Matter Disease, GM3 Gangliosidosis, PKAN, Tay-Sachs Disease, NP Deficiency, Osteopetrosis, Alpha-Mannosidosis, Sandhoff Disease, Niemann-Pick Diseases, MPS IV, Gaucher Disease, GAN, GM1 Gangliosidoses, Morquio Disease, S-Adenosylhomocysteine Hydrolase Deficiency, Batten Disease, Pelizaeus-Merzbacher Disease, Leukodystrophy, Lysosomal Storage Diseases, Purine Nucleoside Phosphorylase Deficiency, Multiple Sulfatase Deficiency Disease
Interventions: Palliative Care, Hematopoetic Stem Cell Transplantation; Other · Biological
Lead sponsor: University of Pittsburgh; Other
Eligibility: Not listed
Enrollment: 1,500 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2035
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Severe Combined Immunodeficiency (SCID), Immunodeficiency With Predominant T-cell Defect, Unspecified, Severe Chronic Neutropenia, Chronic Granulomatous Disease (CGD), Hyper IgE Syndromes, Hyper IgM Deficiencies, Wiskott-Aldrich Syndrome, Mendelian Susceptibility to Mycobacterial Disease, Common Variable Immune Deficiency (CVID)
Interventions: CD3/CD19 negative allogeneic hematopoietic stem cells; Biological
Lead sponsor: Paul Szabolcs; Other
Eligibility: 5 Years to 45 Years
Enrollment: 16 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2027
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

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: Microbial Colonization, Extreme Prematurity, Early-Onset Neonatal Sepsis, Late-Onset Neonatal Sepsis, Necrotizing Enterocolitis of Newborn, Death; Neonatal
Interventions: Ampicillin, Gentamycin, Normal saline; Drug
Lead sponsor: Michael Morowitz; Other
Eligibility: Up to 4 Hours
Enrollment: 359 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2026
U.S. locations: 14
States / cities: Mobile, Alabama • San Diego, California • New Haven, Connecticut + 10 more

Conditions: Mucopolysaccharidosis II
Interventions: AGT-182; Drug
Lead sponsor: ArmaGen, Inc; Industry
Eligibility: 18 Years and older · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2017
U.S. locations: 3
States / cities: Oakland, California • Orange, California • Decatur, Georgia

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Healthy Outcomes of Pregnancy Education

CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)

Maternal Hyperoxygenation for Intrapartum Fetal Heart Rate Tracing Abnormalities

Get Social Media and Risk-Reduction Training

Inflammatory Cytokines Associated With Perinatal Brain Injury

Generations in Families Talking Safe Sleep

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

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

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

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

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)

Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II

Improving Pediatrician Counseling About Infant Safe Sleep Using the Electronic Medical Record

An Observational Study Evaluating Anti-Idursulfase Serum Antibody Response in Hunter Syndrome Patients

Extension of Study TKT024 Evaluating Long-Term Safety and Clinical Outcomes in MPS II Patients Receiving Idursulfase

The Happiest Baby, Inc. SNOO Postmarket Surveillance Study

Clinical Trial of Growth Hormone in MPS I, II, and VI

Premature Infants Receiving Milking or Delayed Cord Clamping: PREMOD2

Longitudinal Study of Neurodegenerative Disorders

Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases

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

NICU Antibiotics and Outcomes Trial

Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter Syndrome