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Results for “Sphingolipidoses”

551 trials

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Showing 20 of 551 results

Large-scale testing (Phase 3)Study completedNCT00478647
What this trial is testing

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

Who this might be right for
Gaucher Disease
Shire 40
Not applicableUnknownNCT02000310
What this trial is testing

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage Disorders
O & O Alpan LLC 80
Not applicableEnded earlyNCT04008849
What this trial is testing

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 3
Testing effectiveness (Phase 2)WithdrawnNCT02715505
What this trial is testing

Safety and Exploratory Efficacy of HSC835 in Patients With Inherited Metabolic Disorders (IMD)

Who this might be right for
Inherited Metabolic Disorders IMD
Novartis Pharmaceuticals
Not applicableStudy completedNCT04804566
What this trial is testing

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Who this might be right for
Fabry Disease
Amicus Therapeutics 44
Not applicableLooking for participantsNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Post-approval studies (Phase 4)Ended earlyNCT00230607
What this trial is testing

Study of the Effects of Fabrazyme Treatment on Lactation and Infants

Who this might be right for
Fabry DiseaseAlpha Galactosidase A Deficiency
Genzyme, a Sanofi Company 7
Not applicableStudy completedNCT00233870
What this trial is testing

A Long Term Safety and Efficacy Study of Fabrazyme Replacement Therapy in Japanese Patients With Fabry Disease.

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 405
Testing effectiveness (Phase 2)Study completedNCT04455230
What this trial is testing

A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190

Who this might be right for
Fabry DiseaseLysosomal Storage Diseases
Spur Therapeutics 3
Not applicableStudy completedNCT05186324
What this trial is testing

Fabry Patient's Experience Of PegunigaLsidasE Alfa Monthly Infusion

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 23
Not applicableUnknownNCT05914727
What this trial is testing

Biomarkers Of Lung InVolvement In ASMD

Who this might be right for
Asmd, Visceral Type
Eline C. B. Eskes 68
Not applicableStudy completedNCT00001289
What this trial is testing

Effects of Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 70
Not applicableLooking for participantsNCT07382128
What this trial is testing

Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes

Who this might be right for
HCM - Hypertrophic CardiomyopathyAnderson Fabry DiseaseCardiac Magnetic Resonance Imaging+1 more
IRCCS Azienda Ospedaliero-Universitaria di Bologna 250
Post-approval studies (Phase 4)WithdrawnNCT00312767
What this trial is testing

A Study in Patients With Fabry Disease Who Are on Chronic Hemodialysis Therapy for Treatment of End-stage Renal Insufficiency.

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company
Not applicableLooking for participantsNCT03362164
What this trial is testing

Evaluation of HEArt invoLvement in Patients With FABRY Disease

Who this might be right for
Rare DiseasesFabry DiseaseFabry Disease, Cardiac Variant+1 more
Wuerzburg University Hospital 650
Large-scale testing (Phase 3)Study completedNCT03614234
What this trial is testing

Open Label Extension of 2 mg/kg Pegunigalsidase Alfa (PRX-102) Every 4 Weeks in Adult Fabry Disease Patients

Who this might be right for
Fabry Disease
Chiesi Farmaceutici S.p.A. 29
Not applicableStudy completedNCT03230149
What this trial is testing

Prevalence and Characteristics of Fabry Disease (FD) in Patients With Stroke or Small Fiber Neuropathy

Who this might be right for
Fabry Disease
Assistance Publique - Hôpitaux de Paris 379
Not applicableLooking for participantsNCT03190837
What this trial is testing

A Long-term Follow-up Study of Gaucher Disease

Who this might be right for
Gaucher Disease
Duke University 200
Testing effectiveness (Phase 2)Study completedNCT00068107
What this trial is testing

Dosing Study of Replagal in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Baylor Research Institute 13
Not applicableLooking for participantsNCT03333200
What this trial is testing

Longitudinal Study of Neurodegenerative Disorders

Who this might be right for
MLDKrabbe DiseaseALD+24 more
University of Pittsburgh 1,500
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