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Lysosomal Storage Diseases

Clinical trial condition

Lysosomal Storage Diseases

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

710 public trials 130 recruiting studies

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Lysosomal Storage Diseases trial results

Completed

Natural History Studies of Mucopolysaccharidosis III

NCT02037880

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +11 more
Locations: Nationwide Children's Hospital Columbus, Ohio, United States

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Completed

The Functional Consequences of the CTNS-deletion for the TRPV1-receptor in Cystinosis Patients

NCT02533076

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Cystinosis, Genetic Diseases, Inborn, +4 more
Locations: Centre for Clinical Pharmacology, UZ Leuven Leuven, Vlaams-Brabant, Belgium

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Completed

Screening an Orthopedic Population for Mildly-affected Individuals With Morquio Syndrome A and Maroteaux-Lamy Syndrome

NCT01961518

Conditions: Carbohydrate Metabolism, Inborn Errors, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Connective Tissue Diseases, +14 more
Locations: Greenwood Genetic Center Greenville, South Carolina, United States

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Completed

Diagnosis of Mucopolysaccharidosis Disorders in Patients Presenting With Bilateral Hip Disease

NCT01707433

Conditions: Bone Diseases, Bone Diseases, Developmental, Carbohydrate Metabolism, Inborn Errors, +15 more
Locations: Children's Hospitals and Clinics of Minnesota Minneapolis, Minnesota, United States

Gillette Children's Specialty Healthcare Saint Paul, Minnesota, United States

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Completed

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

NCT01884220

Conditions: Acid Cholesteryl Ester Hydrolase Deficiency, Type 2, Cholesterol Ester Storage Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, +10 more
Locations: Cincinnati Children's Hospital Medical Center Cincinnati, Ohio, United States

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Completed

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

NCT01136304

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Tower Cancer Research Foundation Beverly Hills, California, United States

Northwest Oncology Hematology Associates PA Coral Springs, Florida, United States

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Completed

Taste Evaluation of Different Liquid Formulations With Eliglustat

NCT02422654

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Investigational Site Number 840001 Evansville, Indiana, United States

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Completed

A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

NCT00140621

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Fujita Health University Hospital Aichi, Japan

Sapporo Medical University Hospital Hokkaido, Japan

Akune Citizen Hospital Kagoshima, Japan

Tohoku University Hospital Miyagi, Japan

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Completed

A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.

NCT00233870

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Tohoku University Hospital Sendai, Japan

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Completed

A Prospective, Observational Study in Patients With Late-Onset Pompe Disease

NCT00077662

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +12 more
Locations: Children's Hospital Medical Center Washington D.C., District of Columbia, United States

School of Medicine, Campus Box 8111 St Louis, Missouri, United States

Children's Hospital & Regional Medical Center Seattle, Washington, United States

Institut de Myologie, Groupe Hospitalier Pitie-Salpetriere, Batimant Babinski Paris, France

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Lysosomal Storage Diseases

Lysosomal Storage Diseases trial results

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