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Clinical trial condition

Sphingolipidoses

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

390 public trials 81 recruiting studies
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Sphingolipidoses trial results

Completed

Multicenter Study of HGT-1110 Administered Intrathecally in Children With Metachromatic Leukodystrophy (MLD)

NCT01510028

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • The Children's Hospital at Westmead Westmead, Australia
  • Rigshospitalet Copenhagen, Denmark
  • Hopital de Bicetre Le Kremlin-Bicêtre, Île-de-France Region, France
  • Center for Pediatric Clinical Studies Tübingen, Baden-Wurttemberg, Germany
View Trial Details
Completed

Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

NCT01842841

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations
  • Hamamatsu University School of Medicine Hamamatsu, Shizuoka, Japan
  • Iwata City Hospital Ōkubo, Shizuoka, Japan
  • The Jikei University School of Medicine Minato-ku, Tokyo, Japan
  • Chiba Children's Hospital Chiba, Japan
View Trial Details
Completed

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

NCT01685216

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Alexandria University Hospital Alexandria, Egypt
  • Abu El Rich Hospital, Cairo University Hospital Cairo, Egypt
  • Children's Hospital, Ain Shams University Hospital Cairo, Egypt
  • KEM Hospital Research Centre Pune, Maharashtra, India
View Trial Details
Completed

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

NCT00635427

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations
  • Los Angeles Medical Center Los Angeles, California, United States
  • Children's Hospital Oakland Oakland, California, United States
  • Emory University School of Medicine Atlanta, Georgia, United States
  • Children's Memorial Hospital Chicago, Illinois, United States
View all 21 locations
View Trial Details
Completed

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

NCT00478647

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations
  • Regional Metabolic Center Los Angeles, California, United States
  • Children's Hospital Oakland Oakland, California, United States
  • Emory University Decatur, Georgia, United States
  • Feinberg School of Medicine Chicago, Illinois, United States
View all 15 locations
View Trial Details
Completed

Safety Study of Replagal® Therapy in Children With Fabry Disease

NCT01363492

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Emory Division of Medical Genetics Decatur, Georgia, United States
  • Duke University Medical Center Durham, North Carolina, United States
  • Baylor University Medical Center Dallas, Texas, United States
  • University of Utah Hospital Salt Lake City, Utah, United States
View Trial Details
Completed

Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease

NCT00864851

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • AKDHC Tucson Access Center Tucson, Arizona, United States
  • University of Iowa Hospitals and Clinics Iowa City, Iowa, United States
  • New York Unversity School of Medicine New York, United States
  • O & O Alpan, LLC Springfield, Virginia, United States
View Trial Details
Completed

Extension Study of TKT028 Evaluating Safety and Clinical Outcomes of Replagal® in Adult Patients With Fabry Disease

NCT01124643

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • University of Iowa Hospitals and Clinics Iowa City, Iowa, United States
  • O&O Alpan LLC Fairfax, Virginia, United States
  • The Royal Melbourne Hospital Parkville, Australia
  • 1st School of Medicine Charles University Prague, Czechia
View Trial Details
Completed

A Multicenter Open-Label Treatment Protocol to Observe the Safety of Replagal (Agalsidase Alfa) Enzyme Replacement Therapy in Canadian Patients With Fabry Disease

NCT01298141

Conditions
Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations
  • Alberta Children's Hospital Calgary, Alberta, Canada
  • University of Alberta Hospital Edmonton, Alberta, Canada
  • Vancouver General Hospital Vancouver, British Columbia, Canada
  • University of Manitoba Winnipeg, Manitoba, Canada
View Trial Details

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