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Conditions: Mucopolysaccharidoses
Interventions: Not listed
Lead sponsor: University of Minnesota; Other
Eligibility: 3 Years to 18 Years
Enrollment: 1,147 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2017
U.S. locations: 2
States / cities: Orange, California • Minneapolis, Minnesota

Conditions: SIDS, Infant Development
Interventions: Safe; Behavioral
Lead sponsor: Milton S. Hershey Medical Center; Other
Eligibility: Up to 14 Days
Enrollment: 130 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2019
U.S. locations: 1
States / cities: Hershey, Pennsylvania

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: Breastfeeding, SUID, SIDS
Interventions: Prenatal Safe Sleep Mobile Health (mHealth) Messaging, Prenatal Breastfeeding Mobile Health (mHealth) Messaging, Postnatal Safe Sleep Mobile Health (mHealth) Messaging, Postnatal Breastfeeding Mobile Health (mHealth) Messaging; Behavioral
Lead sponsor: Boston University; Other
Eligibility: 14 Years and older · Female only
Enrollment: 2,093 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 1
States / cities: Boston, Massachusetts

Conditions: Hunter Syndrome
Interventions: Elaprase for intravenous (IV) infusion; Drug
Lead sponsor: Shire; Industry
Eligibility: Up to 5 Years · Male only
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2025
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Sudden Infant Death Syndrome
Interventions: Lifestyle Counseling; Other
Lead sponsor: University of Virginia; Other
Eligibility: 18 Years and older · Female only
Enrollment: 616 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2004 – 2015
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

Conditions: Hunter Syndrome
Interventions: No treatment; Other
Lead sponsor: Shire; Industry
Eligibility: Up to 70 Years · Male only
Enrollment: 10 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2012 – 2013
U.S. locations: 5
States / cities: Decatur, Georgia • Chicago, Illinois • Minneapolis, Minnesota + 2 more

Conditions: Mucopolysaccharidosis (MPS), Hunter Syndrome
Interventions: ELAPRASE, Rituximab, Methotrexate, Intravenous Immunoglobulin (IVIG); Drug
Lead sponsor: Takeda; Industry
Eligibility: Up to 6 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 8
States / cities: Phoenix, Arizona • Oakland, California • San Diego, California + 5 more

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Pompe Disease Infantile-Onset, Neuronopathic Gaucher Disease, Wolman Disease
Interventions: There is no intervention; Other
Lead sponsor: University of California, San Francisco; Other
Eligibility: Up to 64 Years
Enrollment: 250 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2050
U.S. locations: 1
States / cities: San Francisco, California

Conditions: SIDS
Interventions: JessieHug; Device
Lead sponsor: Weill Medical College of Cornell University; Other
Eligibility: 0 Months to 4 Months
Enrollment: 31 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2026
U.S. locations: 1
States / cities: New York, New York

Conditions: Health Behavior
Interventions: Reduce Infant Mortality; Behavioral
Lead sponsor: University of Virginia; Other
Eligibility: 18 Years and older
Enrollment: 1,200 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2011 – 2015
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

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
Interventions: Neurobehavioral testing, Visual and auditory assessments; Behavioral · Other
Lead sponsor: Shire; Industry
Eligibility: 32 Months to 18 Years · Male only
Enrollment: 33 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2009 – 2011
U.S. locations: 1
States / cities: Chapel Hill, North Carolina

Conditions: MPS II, Mucopolysaccharidosis II, Hunter Syndrome
Interventions: Not listed
Lead sponsor: Duke University; Other
Eligibility: Not listed
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2024
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Mucopolysaccharidosis (MPS), Hunter Syndrome
Interventions: Not listed
Lead sponsor: Shire; Industry
Eligibility: 2 Years to 18 Years · Male only
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2013 – 2016
U.S. locations: 3
States / cities: Oakland, California • Chicago, Illinois • Chapel Hill, North Carolina

Conditions: Hunter Syndrome
Interventions: Not listed
Lead sponsor: Shire; Industry
Eligibility: Not listed
Enrollment: 1,443 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2023
U.S. locations: 1
States / cities: Lexington, Massachusetts

Conditions: Adrenoleukodystrophy, Batten Disease, Mucopolysaccharidosis II, Leukodystrophy, Globoid Cell, Leukodystrophy, Metachromatic, Neimann Pick Disease, Pelizaeus-Merzbacher Disease, Sandhoff Disease, Tay-Sachs Disease, Brain Diseases, Metabolic, Inborn, Alpha-Mannosidosis, Sanfilippo Mucopolysaccharidoses
Interventions: DUOC-01; Biological
Lead sponsor: Joanne Kurtzberg, MD; Other
Eligibility: 1 Week to 22 Years
Enrollment: 40 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2027
U.S. locations: 1
States / cities: Durham, North Carolina

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: Mucopolysaccharidosis II
Interventions: tividenofusp alfa, idursulfase; Drug
Lead sponsor: Denali Therapeutics Inc.; Industry
Eligibility: 2 Years to 25 Years
Enrollment: 63 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2027
U.S. locations: 8
States / cities: Oakland, California • Chicago, Illinois • Hackensack, New Jersey + 5 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: 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: 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: 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: Mucopolysaccharidosis II
Interventions: Long-term Follow-Up; Other
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: Male only
Enrollment: 27 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2028
U.S. locations: 3
States / cities: San Francisco, California • New Brunswich, New Jersey • Philadelphia, Pennsylvania

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Carotid Structure and Function in MPS Syndromes: A Multicenter Study of the Lysosomal Disease Network

Improving Infant Sleep Safety With the Electronic Health Record

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

Social Media and Risk Reduction Teaching-Enhanced Reach

Long-term Evaluation on Height and Weight in Patients With MPS II Who Started Treatment at < 6 Years of Age

Factors Influencing the Racial Disparity in Sudden Infant Death Syndrome (SIDS)

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome

A Study of ELAPRASE in Treatment-naïve Participants With Hunter Syndrome (Mucopolysaccharidosis [MPS] II)

Registry of Patients Diagnosed With Lysosomal Storage Diseases

JessieHug Feasibility and Usability Assessment

Reduce African-American Infant Mortality

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

Screening Study to Identify Pediatric Patients With Hunter Syndrome Who Demonstrate Evidence of Central Nervous System (CNS) Involvement and Who Are Currently Receiving Treatment With Elaprase®

MPS II Immunophenotyping

Observational Study to Evaluate Neurodevelopmental Status in Pediatric Patients With Hunter Syndrome (MPS II)

Hunter Outcome Survey (HOS)

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

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

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

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

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

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

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

Long-term Follow-Up for RGX-121