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Metachromatic Leukodystrophy

Clinical trial condition

Metachromatic Leukodystrophy

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

18 public trials 2 recruiting studies

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Metachromatic Leukodystrophy trial results

Recruiting

The Myelin Disorders Biorepository Project

NCT03047369

Conditions: 4H Syndrome, ADLD, AGS, +173 more
Locations: Children's Hospital of Los Angeles Los Angeles, California, United States

Children's Hospital of Orange County Orange, California, United States

Stanford University (Lucile Packard Children's Hospital) Palo Alto, California, United States

University of California, Davis (UC Davis Health) Sacramento, California, United States

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Recruiting

Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

NCT04925349

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +37 more
Locations: AP-HP Hôpital Bicêtre Le Kremlin-Bicêtre, France

AP-HP Hôpital La Pitié Salpêtrière Paris, France

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Enrolling by Invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

NCT05368038

Conditions: Acid Sphingomyelinase Deficiency, Brain Diseases, Brain Diseases, Metabolic, +65 more
Locations: Maimonides Medical Center Brooklyn, New York, United States

NYU Langone Hospital - Brooklyn Brooklyn, New York, United States

North Shore University Hospital Manhasset, New York, United States

NYU Langone Health - Tisch Hospital New York, United States

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Completed

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

NCT01303146

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Department of Pediatric Endocrinology and Neurology, Saint Vincent de Paul Hospital Paris, France

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Completed

A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

NCT03392987

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations: Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) Milan, Italy

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Completed

Intracerebral Gene Therapy for Children With Early Onset Forms of Metachromatic Leukodystrophy

NCT01801709

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Bicêtre Hospital - Paris Sud Le Kremlin-Bicêtre, France

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Completed

Gene Therapy for Metachromatic Leukodystrophy (MLD)

NCT01560182

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +20 more
Locations: Ospedale San Raffaele - Telethon Institute for Gene Therapy (OSR-TIGET) Milan, Italy

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Completed

LeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for Leukodystrophies

NCT02699190

Conditions: 4H Syndrome, ADLD, AGS, +173 more
Locations: The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, 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

The Natural History of Metachromatic Leukodystrophy Study (HOME Study)

NCT04628364

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
Locations: National Organization for Rare Disorders Danbury, Connecticut, United States

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Metachromatic Leukodystrophy

Metachromatic Leukodystrophy trial results

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