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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Large-scale testing (Phase 3)UnknownNCT06081062
What this trial is testing

Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

Who this might be right for
Fabry Disease
ISU Abxis Co., Ltd. 24
Not applicableUnknownNCT05719337
What this trial is testing

Multi-Modality Echocardiographic Techniques in Pathological Left Ventricular Hypertrophy Adults

Who this might be right for
Left Ventricular Hypertrophy
First Hospital of China Medical University 660
Not applicableStudy completedNCT05671770
What this trial is testing

Study Based on Electronic Health RecOrds to Identify Patients at High-risk of Fabry DiseasE (HOPE Fabry)

Who this might be right for
Fabry Disease
Sanofi 15
Large-scale testing (Phase 3)Active Not RecruitingNCT05280548
What this trial is testing

Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease

Who this might be right for
Fabry Disease
Sanofi 104
Not applicableNot Yet RecruitingNCT07046338
What this trial is testing

Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

Who this might be right for
Metachromatic Leukodystrophy (MLD)
Shenzhen Geno-Immune Medical Institute 10
Large-scale testing (Phase 3)Active Not RecruitingNCT04020055
What this trial is testing

Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 14
Not applicableUnknownNCT04943991
What this trial is testing

Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score

Who this might be right for
Fabry DiseaseFabry Disease, Cardiac VariantLysosomal Storage Diseases+2 more
Wuerzburg University Hospital 200
Not applicableLooking for participantsNCT05586243
What this trial is testing

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

Who this might be right for
Gaucher Disease, Type 3
University of Minnesota 5
Large-scale testing (Phase 3)Study completedNCT02194985
What this trial is testing

Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Amicus Therapeutics 84
Not applicableWithdrawnNCT01093105
What this trial is testing

An Observational Study of Pediatric Subjects With Globoid Cell Leukodystrophy (GLD)

Who this might be right for
Leukodystrophy, Globoid Cell
Shire
Testing effectiveness (Phase 2)Ended earlyNCT00681811
What this trial is testing

Open-Label Extension Study of Recombinant Human Arylsulfatase A (HGT-1111) in Late Infantile MLD

Who this might be right for
Late Infantile Metachromatic Leukodystrophy
Shire 11
Large-scale testing (Phase 3)Study completedNCT00074971
What this trial is testing

The Safety and Efficacy of Fabrazyme in Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 58
Not applicableStudy completedNCT04106544
What this trial is testing

A Prospective and Retrospective Cohort Study in Patients With Chronic Forms of Acid Sphingomyelinase Deficiency (ASMD)

Who this might be right for
Sphingomyelin Lipidosis
Sanofi 84
Not applicableWithdrawnNCT00639132
What this trial is testing

The Natural History of Metachromatic Leukodystrophy

Who this might be right for
Metachromatic Leukodystrophy
University of Pittsburgh
Not applicableStudy completedNCT00983879
What this trial is testing

The Natural History of Infantile Globoid Cell Leukodystrophy

Who this might be right for
Infantile Globoid Cell Leukodystrophy
Zymenex A/S 6
Not applicableStudy completedNCT04602364
What this trial is testing

French Prospective, Observational Cohort Study of Patients With Fabry Disease Treated With Migalastat

Who this might be right for
Fabry Disease
Amicus Therapeutics France SAS 48
Large-scale testing (Phase 3)Study completedNCT00891202
What this trial is testing

Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

Who this might be right for
Gaucher Disease, Type 1
Genzyme, a Sanofi Company 40
Testing effectiveness (Phase 2)Study completedNCT00045812
What this trial is testing

SCH-58235 (Ezetimibe) to Treat Homozygous Sitosterolemia

Who this might be right for
Lipoidosis
National Heart, Lung, and Blood Institute (NHLBI) 5
Not applicableNot Yet RecruitingNCT06985212
What this trial is testing

National Multicentre Study of the Natural History of Acid Sphingo-myelinase Deficiency in Adults and Children

Who this might be right for
Acid Sphingomyelinase Deficiency (ASMD)Niemann Pick Disease
Wladimir MAUHIN, Dr 200
Not applicableStudy completedNCT01198899
What this trial is testing

Belgian Screening Project for the Detection of Anderson-Fabry Disease in Hypertrophic Cardiomyopathy

Who this might be right for
Left Ventricular Hypertrophy
University Hospital, Ghent 540
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