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Clinical trial condition

Lysosomal Storage Diseases

Explore clinical trials studying Lysosomal Storage Diseases. Study availability and eligibility vary by location and protocol.

77 public trials 13 recruiting studies
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Lysosomal Storage Diseases trial results

Completed

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

NCT03500094

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Clinical Study Site Tampa, Florida, United States
  • Clinical Study Site Atlanta, Georgia, United States
  • Clinical Study Site Minneapolis, Minnesota, United States
  • Clinical Study Site Columbia, Missouri, United States
View Trial Details
Completed

Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome)

NCT01415427

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +12 more
Locations
  • Phoenix, Arizona, United States
  • Oakland, California, United States
  • Orange, California, United States
  • Wilmington, Delaware, United States
View all 43 locations
View Trial Details
Completed

A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®

NCT00920647

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +23 more
Locations
  • University of North Carolina at Chapel Hill Chapel Hill, North Carolina, United States
  • Birmingham Children's Hospital Birmingham, United Kingdom
  • Birmingham Children's Hospital NHS Foundation Trust Birmingham, United Kingdom
View Trial Details
Completed

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

NCT02055118

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +23 more
Locations
  • Children's Hospital and Research Center at Oakland Oakland, California, United States
  • Ann & Robert H Lurie Children's Hospital of Chicago Chicago, Illinois, United States
  • University of North Carolina Chapel Hill, North Carolina, United States
  • Women's and Children's Hospital, 72 King William Road North Adelaide, Australia
View Trial Details
Completed

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

NCT00630747

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +24 more
Locations
  • St. Joseph's Hospital Phoenix, Arizona, United States
  • Pediatric Clinical Research Center, Children's Hospital Oakland Oakland, California, United States
  • The Children's Hospital Denver, Colorado, United States
  • Harbin Clinic Rome, Georgia, United States
View all 52 locations
View Trial Details
Completed

Safety and Clinical Outcomes in Hunter Syndrome Patients 5 Years of Age and Younger Receiving Idursulfase Therapy

NCT00607386

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +24 more
Locations
  • Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica Porto Alegre, Rio Grande do Sul, Brazil
  • Instytut Pomnik Centrum Zdrowia Dziecka, Klinika Chorob Metaboliczynch, Endokrynologii i Diabetologii Warsaw, Poland
  • National Taiwan University Hospital, Dept. of Pediatrics and Medical Genetics Taipei, Taiwan
View Trial Details
Completed

Randomized, Controlled, Open-label, Multicenter, Safety and Efficacy Study of rhHNS Administration Via an IDDD in Pediatric Patients With Early Stage MPS IIIA Disease

NCT02060526

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +18 more
Locations
  • Los Angeles Biomedical Research Torrance, California, United States
  • University of Minnesota Department of Pediatrics Minneapolis, Minnesota, United States
  • University of North Carolina Chapel Hill, North Carolina, United States
  • Hospital Universitario Austral A Unidad de Investigacion Buenos Aires, Argentina
View Trial Details
Completed

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

NCT00882921

Conditions
Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +23 more
Locations
  • Children's Hospital & Research Center Oakland Oakland, California, United States
  • Children's Hospitals and Clinics of Minnesota, Division of Genetics Minneapolis, Minnesota, United States
  • Hospital de Clinicas de Porto Alegre, Servico de Genetica Medica Porto Alegre, Rio Grande do Sul, Brazil
  • Birmingham Children's Hospital Birmingham, United Kingdom
View Trial Details
Completed

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

NCT02416661

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations
  • University Hospital Center Mother Teresa Tirana, Albania
  • Aristotle University of Thessaloniki, Ippokration General Hospital Thessaloniki, Greece
  • Centre for Human Genetics Bangalore, India
  • Shaare Zedek Medical Center Jerusalem, Israel
View Trial Details

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