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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

Not yet recruiting

Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

NCT07479953

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +18 more
Locations: Not specified

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Not yet recruiting

A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease

NCT07778667

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Not specified

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Not yet recruiting

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

NCT07740512

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +24 more
Locations: Not specified

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Not yet recruiting

Study to Evaluate the Efficacy and Safety of Nizubaglustat (AZ-3102) in Patients With Gaucher Disease Type 3 (GD3)

NCT07715084

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: SMS Medical College Jaipur Jaipur, India

Maula a Azad medical college Delhi New Delhi, India

Sir Ganga Ram Hospital New Delhi, India

University of Child Health Sciences and the Children's Hospital Lahore, Pakistan

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Not yet recruiting

A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease

NCT07603050

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +15 more
Locations: Not specified

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Not yet recruiting

A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion

NCT05669729

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +14 more
Locations: Not specified

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Not yet recruiting

Care Pathway for Patients With Fabry's Disease (Fabry-PATH)

NCT07485660

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Service de Cardiologie - 1 Avenue du Professeur Jean Poulhès Toulouse, Occitanie, France

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Not yet recruiting

Translational Potential of ex Vivo Gene Therapy in GM2 Gangliosidosis

NCT07445490

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +16 more
Locations: Département de Neurologie Paris, France

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Not yet recruiting

Novel Diagnostic and Prognostic Predictors in Fabry Cardiomyopathy: Proof of Concept in a Rare Disease

NCT07351136

Conditions: Brain Diseases, Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, +19 more
Locations: Not specified

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Not yet recruiting

Pediatric Arthropathy Beyond Inflammation: Clinical Spectrum and Diagnostic Approach at Assiut University Children Hospital

NCT07173010

Conditions: Arthropathy, progressive pseudorheumatoid, of childhood, Bone Diseases, Bone Diseases, Developmental, +36 more
Locations: Not specified

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