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Sulfatidosis

Clinical trial condition

Sulfatidosis

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

37 public trials 12 recruiting studies

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Sulfatidosis trial results

Recruiting

Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.

NCT04880356

Conditions: 2-Hydroxyglutaricaciduria, Adrenal Gland Diseases, Adrenal Insufficiency, +58 more
Locations: Fondazione IRCCS Istituto Neurologico Carlo Besta Milan, Milano, Italy

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Recruiting

Safety and Efficacy of SMART101 in Adult Patients With Hematological Malignancies After Haploidentical HSCT With Post-transplant Cyclophosphamide

NCT05768035

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
Locations: Institut Paoli Calmettes Marseille, France

Centre hospitalier universitaire de Nantes Nantes, France

Hôpital Saint-Louis Paris, France

CHU Toulouse- Institut Universitaire du cancer Toulouse- Oncopole Toulouse, 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

A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy

NCT03771898

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Los Angeles Biomedical Research Institute at Harbor-UCLA Torrance, California, United States

Childrens Hospital Colorado Aurora, Colorado, United States

Rare Disease Research, LLC Atlanta, Georgia, United States

Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States

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Completed

An Efficacy and Safety Study of HGT-1110 in Participants With Metachromatic Leukodystrophy

NCT01887938

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: The Children's Hospital at Westmead Westmead, Australia

Hospital de Clinicas de Porto Alegre Porto Alegre, Brazil

Detska Interni Klinika, Lf Mu A Fn Brno Brno, Czechia

Rigshospitalet Copenhagen, Denmark

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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

Imaging Study of the White Matter Lesions in Children With Metachromatic Leucodystrophy

NCT01325025

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Unité de recherche biomédicale, Neurospin, I2BM / DSV / SAC/ CEA, Gif-sur-Yvette, France

Service de Neurologie Pédiatrique, Hôpital Bicêtre Paris, France

Bâtiment Lavoisier - Unité INSERM U 663,Hôpital Necker Enfants Malades Paris, France

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Sulfatidosis

Sulfatidosis trial results

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