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Mucopolysaccharidoses

Clinical trial condition

Mucopolysaccharidoses

Explore clinical trials studying Mucopolysaccharidoses. Study availability and eligibility vary by location and protocol.

192 public trials 30 recruiting studies

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Mucopolysaccharidoses trial results

Completed

North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2

NCT03548779

Conditions: Autism Spectrum Disorder, Brain Diseases, Brain Malformation, +55 more
Locations: Mission Health Asheville, North Carolina, United States

University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States

East Carolina University Greenville, North Carolina, United States

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Completed

A Study of JR-171 in Patients With Mucopolysaccharidosis I

NCT04227600

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
Locations: UCSF Benioff Children's Hospital Oakland Oakland, California, United States

Hospital de Clínicas de Porto Alegre Porto Alegre, Brazil

Instituto de Genética e Erros Inatos do Metabolismo - IGEIM São Paulo, Brazil

Fukuoka Children's Hospital Fukuoka, Japan

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Completed

A Natural History Study of Biomarkers and Clinical Outcomes in Mucopolysaccharidosis Type IIIA (MPS IIIA; Sanfilippo Syndrome)

NCT05523206

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: Sanguine Biosciences, Inc. Woburn, Massachusetts, United States

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Completed

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

NCT04571970

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +23 more
Locations: University of California San Francisco, Benioff Children's Hospital Oakland, California, United States

McGill University Heath Center Montreal, Quebec, Canada

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Completed

A Study of Elaprase in Children and Adults With Hunter Syndrome (Mucopolysaccharidosis II) in India

NCT05058391

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations: SAT Hospital - Govt Medical College Thiruvananthapuram, Kerala, India

JK Lone Hospital Jaipur, Rajasthan, India

Institute of Child Health Kolkata, India

All India Institute of Medical Sciences (AIIMS) New Delhi, India

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Completed

CO2 Supplement for Treatment of Acute Mountain Sickness

NCT06393998

Conditions: Acute Mountain Sickness, Altitude Sickness, Apnea, +25 more
Locations: Guangzhou Medical University Guangzhou, Guangdong, China

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Completed

Gene Therapy in Patients With Mucopolysaccharidosis Disease

NCT03173521

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +10 more
Locations: Department of Translational Medicine (DISMET) of "Federico II" University, Naples Naples, Italy

Children's Hospital Hacettepe University Ankara, Turkey (Türkiye)

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Completed

Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products

NCT00695279

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +31 more
Locations: St. Jude Children's Research Hospital Memphis, Tennessee, United States

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Completed

Hunter Outcome Survey (HOS)

NCT03292887

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +17 more
Locations: Shire Lexington, Massachusetts, United States

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Completed

Open-label Study of Anakinra in MPS III

NCT04018755

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +9 more
Locations: The Lundquist Institute at Harbor-UCLA Medical Center Torrance, California, United States

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Mucopolysaccharidoses

Mucopolysaccharidoses trial results

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