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Neuronal Ceroid-Lipofuscinoses

Clinical trial condition

Neuronal Ceroid-Lipofuscinoses

Explore clinical trials studying Neuronal Ceroid-Lipofuscinoses. Study availability and eligibility vary by location and protocol.

42 public trials 11 recruiting studies

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Neuronal Ceroid-Lipofuscinoses trial results

Active, not recruiting

Gene Therapy Study for Children With CLN5 Batten Disease

NCT05228145

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +10 more
Locations: University of Rochester Rochester, New York, United States

Great Ormond Street Hospital for Children London, United Kingdom

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Completed

Functional Near-Infrared Spectroscopy (fNIRS) Combined With Diffuse Correlation Spectroscopy (DCS) in Neurocognitive Disease as Compared to Healthy Neurotypical Controls

NCT05642221

Conditions: Abnormalities, Multiple, Brain Diseases, Brain Diseases, Metabolic, +33 more
Locations: National Institutes of Health Clinical Center Bethesda, Maryland, United States

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Completed

Gene Therapy For Children With Variant Late Infantile Neuronal Ceroid Lipofuscinosis 6 (vLINCL6) Disease

NCT02725580

Conditions: Ceroid Lipofuscinosis, Neuronal, 6, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +11 more
Locations: Nationwide Children's Hosptial Columbus, Ohio, United States

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Completed

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

NCT05174039

Conditions: Batten Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +10 more
Locations: Texas Children Hospital Houston, Texas, United States

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Completed

Baby Detect : Genomic Newborn Screening

NCT05687474

Conditions: 3-Hydroxy 3-Methyl Glutaric Aciduria, 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency, 3-Hydroxy-3-Methylglutaryl-CoA Synthase 2 Deficiency, +368 more
Locations: CRMN, Hôpital La Citadelle Liège, Wallonia, Belgium

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Completed

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

NCT02678689

Conditions: Batten Disease, CLN2 Disease, CLN2 Disorder, +14 more
Locations: Nationwide Children's Hospital Columbus, Ohio, United States

Universitaetsklinikum Hamburg-Eppendorf Hamburg, Germany

Children's Hospital Bambino Gesù,IRCCS Rome, Piazza, Italy

Great Ormond Street Childrens Hospital London, United Kingdom

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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

An Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 190 in Patients With CLN2 Disease

NCT02485899

Conditions: Batten Disease, CLN2 Disease, CLN2 Disorder, +14 more
Locations: Nationwide Children's Hospital Columbus, Ohio, United States

Universitaetsklinikum Hamburg-Eppendorf Hamburg, Germany

Children's Hospital Bambino Gesù,IRCCS Rome, Piazza, Italy

Great Ormond Street Childrens Hospital London, United Kingdom

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Completed

Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses

NCT04808297

Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System, +9 more
Locations: IRCCS Fondazione Stella Maris Pisa, PI, Italy

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Completed

Safety Study of a Gene Transfer Vector (Rh.10) for Children With Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL)

NCT01161576

Conditions: Batten Disease, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Genetic Diseases, Inborn, +11 more
Locations: Weill Cornell Medical College New York, United States

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Neuronal Ceroid-Lipofuscinoses

Neuronal Ceroid-Lipofuscinoses trial results

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