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NCT03725670
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Lung-Ji Chang Shenzhen, Guangdong, China
NCT04528355
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +119 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
NCT01962415
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +125 more
- Locations
-
- UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States
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
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
NCT02254863
- Conditions
- Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +55 more
- Locations
-
- Duke University Medical Center Durham, North Carolina, United States
NCT07046338
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
-
- Shenzhen Geno-Immune Medical Institute Shenzhen, Guangdong, China
Recruiting
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
NCT01793168
- Conditions
- 1p36 Deletion Syndrome, 3-Methylglutaconic Aciduria, Type V, 4p Deletion Syndrome, Non-Wolf-Hirschhorn Syndrome, +698 more
- Locations
-
- Sanford Health Sioux Falls, South Dakota, United States
- Online Patient Enrollment System Sydney, Australia
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
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
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
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
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
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
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
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
NCT01325025
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
- Locations
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- 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
NCT02699190
- Conditions
- 4H Syndrome, ADLD, AGS, +173 more
- Locations
-
- The Children's Hospital of Philadelphia Philadelphia, Pennsylvania, United States
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
NCT04628364
- Conditions
- Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +23 more
- Locations
-
- National Organization for Rare Disorders Danbury, Connecticut, United States