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Niemann-Pick Diseases

Clinical trial condition

Niemann-Pick Diseases

Explore clinical trials studying Niemann-Pick Diseases. Study availability and eligibility vary by location and protocol.

20 public trials 4 recruiting studies

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Niemann-Pick Diseases trial results

Completed

A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

NCT04845958

Conditions: Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +25 more
Locations: Investigational Site Number 2500036 Amiens, France

Investigational Site Number 2500014 Angers, France

Investigational Site Number 2500034 Argenteuil, France

Investigational Site Number 2500039 Bayonne, France

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Completed

Data Analysis of Adult and Pediatric Participants With Acid Sphingomyelinase Deficiency (ASMD) on Early Access to Olipudase Alfa in France

NCT05359276

Conditions: Acid Sphingomyelinase Deficiency (ASMD), Brain Diseases, Brain Diseases, Metabolic, +19 more
Locations: Investigational site in France France

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Completed

Human Placental-Derived Stem Cell Transplantation

NCT01586455

Conditions: Acute Lymphocytic Leukemia, Acute Myelogenous Leukemia, Adrenal Gland Diseases, +84 more
Locations: Children's Hospital Colorado Denver, Colorado, United States

New York Medical College Valhalla, New York, United States

University of Utah Salt Lake City, Utah, United States

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Completed

Induced Pluripotent Stem Cells for Niemann Pick Disease

NCT03883750

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Childrens Hospital and Institute of Child Health, Ferozepur Road Lahore, Pakistan

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Completed

Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick

NCT04469894

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Icahn School of Medicine at Mount Sinai New York, United States

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Completed

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

NCT01371825

Conditions: Alcohol-Induced Disorders, Alcohol-Related Disorders, Brain Diseases, +33 more
Locations: Irvine, California, United States

Cairo, Egypt

Grenoble, France

Paris, France

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Completed

Stem Cell Transplant for Inborn Errors of Metabolism

NCT00176904

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
Locations: Masonic Cancer Center, University of Minnesota Minneapolis, Minnesota, United States

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Completed

Saccadic Eye Movements in Patients With Niemann-Pick Type C Disease

NCT00316498

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: National Institutes of Health Clinical Center, 9000 Rockville Pike Bethesda, Maryland, United States

Columbia University New York, United States

Oxford GlycoSciences Ltd. Abingdon, United Kingdom

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Completed

Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells

NCT00730314

Conditions: Agranulocytosis, Albinism, Anemia, +77 more
Locations: Children Hospital Los Angeles Los Angeles, California, United States

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Completed

PET Scan of Brain Metabolism in Relation to Age and Disease

NCT00001972

Conditions: Alzheimer Disease, Alzheimer's Disease, Brain Diseases, +32 more
Locations: National Institute of Neurological Disorders and Stroke (NINDS) Bethesda, Maryland, United States

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Niemann-Pick Diseases

Niemann-Pick Diseases trial results

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