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Sphingolipidoses

Clinical trial condition

Sphingolipidoses

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

394 public trials 77 recruiting studies

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Sphingolipidoses trial results

Recruiting

A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease

NCT07187440

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: The First Affiliated Hospital Of USTC (AnHui Provincial Hospital) Hefei, Anhui, China

Peking University First Hospital Beijing, Beijing Municipality, China

Peking University Third Hospital Beijing, Beijing Municipality, China

Peking Union Medical College Hospital Beijing, Beijing Municipality, China

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Recruiting

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

NCT01962415

Conditions: Adrenal Gland Diseases, Adrenal Insufficiency, Adrenoleukodystrophy, +125 more
Locations: UPMC Children's Hospital of Pittsburgh Pittsburgh, Pennsylvania, United States

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Recruiting

Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment

NCT07277361

Conditions: Aged 65 Years or Older, Alpha Galactosidase A Deficiency, Brain Diseases, +22 more
Locations: Groupe Hospitalier Diaconesses Croix Saint-Simon Paris, France

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Recruiting

Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study

NCT07235709

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Division of Cardiology, Yonsei Cardiovascular Hospital, Yonsei University College of Medicine Seoul, South Korea

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Recruiting

A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients

NCT06663358

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: University of Alabama at Birmingham Birmingham, Alabama, United States

Emory University School of Medicine Atlanta, Georgia, United States

Northwestern University - Feinberg School of Medicine - Ann & Robert H. Lurie Children's Hospital of Chicago Chicago, Illinois, United States

University of Iowa Hospitals and Clinics Iowa City, Iowa, United States

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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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Recruiting

A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

NCT04252066

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Amicus Therapeutics, Inc. Pregnancy Registry Philadelphia, Pennsylvania, United States

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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

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Recruiting

the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease

NCT06776419

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +25 more
Locations: Rigshospitalet Copenhagen, Denmark

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Sphingolipidoses

Sphingolipidoses trial results

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