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Showing 49–63 of 63 matching trials from the live ClinicalTrials.gov search.

Filters: Condition: Mucopolysaccharidosis Type II Clear all

Conditions: Hunter Syndrome
Interventions: Idursulfase-IT, Elaprase; Drug
Lead sponsor: Shire; Industry
Eligibility: Up to 18 Years · Male only
Enrollment: 56 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2015 – 2024
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: 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: Acid Sphingomyelinase Deficiency, Ceroid Lipofuscinosis, Neuronal, 2, Cerebrotendinous Xanthomatosis, Fabry Disease, GM1 Gangliosidosis, Gaucher Disease, Lysosomal Acid Lipase Deficiency, Metachromatic Leukodystrophy, Mucopolysaccharidosis II, Mucopolysaccharidosis III-B, Mucopolysaccharidosis IV A, Mucopolysaccharidosis VI, Mucopolysaccharidosis VII, Niemann-Pick Disease, Type C
Interventions: Confirmatory Testing; Diagnostic Test
Lead sponsor: Albert Einstein College of Medicine; Other
Eligibility: Up to 4 Weeks
Enrollment: 100,000 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2021 – 2029
U.S. locations: 9
States / cities: Brooklyn, New York • Manhasset, New York • New York, New York + 2 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: 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: 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: 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: 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
Interventions: data collection; Drug
Lead sponsor: Paul Szabolcs; Other
Eligibility: 2 Months to 60 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2020 – 2028
U.S. locations: 1
States / cities: Pittsburgh, Pennsylvania

Conditions: Mucopolysaccharidosis I, Mucopolysaccharidosis VI, Mannosidosis, Mucolipidosis Type II (I-cell Disease)
Interventions: Stem Cell Transplant, Busulfan, Cyclophosphamide, ATG; Procedure · Drug
Lead sponsor: Masonic Cancer Center, University of Minnesota; Other
Eligibility: Not listed
Enrollment: 41 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 1999 – 2010
U.S. locations: 1
States / cities: Minneapolis, Minnesota

Conditions: Mucopolysaccharidosis II
Interventions: tividenofusp alfa, idursulfase; Drug
Lead sponsor: Denali Therapeutics Inc.; Industry
Eligibility: 2 Years to 25 Years
Enrollment: 65 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 II
Interventions: JR-141; Drug
Lead sponsor: JCR Pharmaceuticals Co., Ltd.; Industry
Eligibility: Not listed
Enrollment: 80 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2028
U.S. locations: 2
States / cities: Oakland, California • Philadelphia, Pennsylvania

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: 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

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Study of Long Term Safety and Clinical Outcomes of Idursulfase IT and Elaprase Treatment in Pediatric Participants Who Have Completed Study HGT-HIT-094

Hunter Outcome Survey (HOS)

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

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

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

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

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

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

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Stem Cell Transplantation for Hurler

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)

An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in MPS II (Hunter Syndrome) Subjects

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

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)