Search | Clinical Trials Finder

Conditions: Mucopolysaccharidosis Disorders, Hurler Syndrome, Hunter Syndrome, Maroteaux Lamy Syndrome, Sly Syndrome, Alpha-Mannosidosis, Fucosidosis, Aspartylglucosaminuria, Glycoprotein Metabolic Disorders, Sphingolipidoses, Recessive Leukodystrophies, Globoid Cell Leukodystrophy, Metachromatic Leukodystrophy, Niemann-Pick B, Niemann-Pick C Subtype 2, Sphingomyelin Deficiency, Peroxisomal Disorders, Adrenoleukodystrophy With Cerebral Involvement, Zellweger Syndrome, Neonatal Adrenoleukodystrophy, Infantile Refsum Disease, Acyl-CoA Oxidase Deficiency, D-Bifunctional Enzyme Deficiency, Multifunctional Enzyme Deficiency, Alpha-methylacyl-CoA Racmase Deficiency, Mitochondrial Neurogastrointestingal Encephalopathy, Severe Osteopetrosis, Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation), Inherited Metabolic Disorders
Interventions: Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen, Osteopetrosis Haploidentical Only Preparative Regimen, cALD SR-A (Standard-Risk, Regimen A), cALD SR-B (Standard-Risk, Regimen B), cALD HR-D (High-Risk, Regimen C), cALD HR-D (High-Risk, Regimen D); Biological · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Up to 55 Years
Enrollment: 149 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2014 – 2029
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Mucopolysaccharidosis Type II (MPS II)
Interventions: RGX-121; Genetic
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 5 Years to 17 Years · Male only
Enrollment: 6 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2021 – 2024
U.S. locations: 1
States / cities: Oakland, California

Conditions: Safe Sleep Education
Interventions: Wearable Blanket (Sleep Sack), Incentive Item; Other
Lead sponsor: University of Kansas; Other
Eligibility: 18 Years and older
Enrollment: 152 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2013 – 2014
U.S. locations: 1
States / cities: Wichita, Kansas

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: 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: 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: 2,126 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 1
States / cities: Boston, Massachusetts

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: Hunter Syndrome
Interventions: Idursulfase-IT; Drug
Lead sponsor: Takeda; Industry
Eligibility: Male only
Healthy volunteers: Healthy volunteers not accepted
U.S. locations: 17
States / cities: Phoenix, Arizona • Oakland, California • Wilmington, Delaware + 14 more

Conditions: Mucopolysaccharidosis II
Interventions: Observational; Other
Lead sponsor: REGENXBIO Inc.; Industry
Eligibility: 1 Month to 8 Years · Male only
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2025
U.S. locations: 2
States / cities: Oakland, California • Philadelphia, Pennsylvania

Conditions: African American Infant Sleep Beliefs and Practices
Interventions: Tailored education, Standard Education; Behavioral
Lead sponsor: University of Michigan; Other
Eligibility: 18 Years and older
Enrollment: 735 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2012 – 2016
U.S. locations: 1
States / cities: Ann Arbor, Michigan

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: 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: 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: 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: 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: Skin Infections, Bacterial
Interventions: Retapamulin Ointment, 1%; Drug
Lead sponsor: GlaxoSmithKline; Industry
Eligibility: 2 Months to 24 Months
Enrollment: 60 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2008
U.S. locations: 6
States / cities: Tuscaloosa, Alabama • Bentonville, Arkansas • Orange, California + 3 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: Sudden Infant Death Syndrome (SIDS)
Interventions: Children's Book, Standard Brochures; Other
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: 15 Years and older
Enrollment: 282 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2014 – 2016
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Sudden Infant Death Syndrome
Interventions: Not listed
Lead sponsor: Rachel Moon, MD; Other
Eligibility: Up to 3 Months
Enrollment: 20 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2009 – 2018
U.S. locations: 1
States / cities: Washington D.C., District of Columbia

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: 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: 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: 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

Search by objective public record fields.

MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

Re-enforcing Safe Sleep Practices for Caregivers With Tangible Incentives

Registry of Patients Diagnosed With Lysosomal Storage Diseases

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

Social Media and Risk Reduction Teaching-Enhanced Reach

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Post-trial Access Program of Idursulfase-IT Along With Elaprase in Children With Hunter Syndrome

Mucopolysaccharidosis Type II Observational

Improving the Self-Efficacy of African American Parents in Infant Supine Sleep

MPS II Immunophenotyping

Generations in Families Talking Safe Sleep

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

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

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

Pharmacokinetics of Retapamulin in Pediatric Subjects With Uncomplicated Skin Infections.

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

Comparing Children's Book to Brochures for Safe Sleep Education in a Home Visiting Program

Changes in Sleep Patterns and Stress in Infants Entering Child Care

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)

Mucopolysaccharidosis Type II Natural History

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

Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome