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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableUnknownNCT04624789
What this trial is testing

Registry Gangliosidoses

Who this might be right for
GangliosidosesGM1 GangliosidosisMorquio B Disease+6 more
SphinCS Lyso Gemeinnutzige UG (Haftungsbeschrankt) 40
Large-scale testing (Phase 3)WithdrawnNCT02921620
What this trial is testing

Study to Evaluate the Safety and EffIcacy of PRX-102 on Gastrointestinal Symptoms in Naïve Fabry Disease

Who this might be right for
Fabry Disease
Protalix
Testing effectiveness (Phase 2)Study completedNCT04046224
What this trial is testing

Dose-Ranging Study of ST-920, an AAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects With Fabry Disease (STAAR)

Who this might be right for
Fabry Disease
Sangamo Therapeutics 36
Not applicableStudy completedNCT02372513
What this trial is testing

National Lysosomal Acid Lipase Deficiency Study

Who this might be right for
Cholesteryl Ester Storage Disease
Ankara University 810
Not applicableUnknownNCT02120235
What this trial is testing

Investigating Lysosomal Storage Diseases in Minority Groups

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more
O & O Alpan LLC 20,000
Testing effectiveness (Phase 2)UnknownNCT00004293
What this trial is testing

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

Who this might be right for
Gaucher's Disease
National Center for Research Resources (NCRR) 24
Early research (Phase 1)WithdrawnNCT00679744
What this trial is testing

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

Who this might be right for
G(M2) GangliosideTay-Sachs Disease GangliosideSandhoff Disease Ganglioside
Exsar Corporation
Not applicableStudy completedNCT01971957
What this trial is testing

Sjogren-Larsson Syndrome: Natural History, Clinical Variation and Evaluation of Biochemical Markers

Who this might be right for
Sjogren-Larsson Syndrome (SLS)
University of Nebraska 20
Not applicableUnknownNCT04758130
What this trial is testing

Getting Global Rare Disease Insights Through Technology Study

Who this might be right for
Metabolic DiseaseFabry DiseaseGaucher Disease+2 more
M.A.G.I.C. Clinic LTD 150
Post-approval studies (Phase 4)Study completedNCT00364858
What this trial is testing

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1Cerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 95
Not applicableLooking for participantsNCT06941025
What this trial is testing

Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding

Who this might be right for
Fabry DiseasePregnancyPregnancy Complications
Chiesi Farmaceutici S.p.A. 10
Post-approval studies (Phase 4)Study completedNCT00074984
What this trial is testing

The Safety and Efficacy of Fabrazyme (Agalsidase Beta) as Compared to Placebo in Patients With Advanced Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 82
Testing effectiveness (Phase 2)Looking for participantsNCT07399704
What this trial is testing

Evaluate the Safety and Efficacy of Nizubaglustat (AZ-3102) in Patients With GM2 Gangliosidosis or Niemann-Pick Type C Disease

Who this might be right for
GM2 GangliosidosisNiemann-Pick Type C Disease
Azafaros B.V. 21
Not applicableUnknownNCT04833595
What this trial is testing

Can Mindful Eating Improve Cholesterol and Glucose Level

Who this might be right for
Cholesterol; Lipidosis
Monash University Malaysia 60
Not applicableStudy completedNCT01019629
What this trial is testing

Fabry Screening Study

Who this might be right for
Fabry Disease
Baylor Research Institute 2,724
Large-scale testing (Phase 3)Study completedNCT05529992
What this trial is testing

Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
Takeda 20
Testing effectiveness (Phase 2)Study completedNCT00975689
What this trial is testing

Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine

Who this might be right for
Niemann-Pick Disease, Type C
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) 35
Testing effectiveness (Phase 2)Study completedNCT00358150
What this trial is testing

The Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

Who this might be right for
Gaucher Disease, Type 1Cerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 26
Testing effectiveness (Phase 2)Study completedNCT03950050
What this trial is testing

Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 40
Not applicableEnded earlyNCT04073888
What this trial is testing

Study of the Spermatic Characteristics of Patients With Fabry Disease

Who this might be right for
Fabry Disease
University Hospital, Bordeaux 22
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