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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT03500094
What this trial is testing

Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Migalastat in Pediatric Subjects (Aged 12 to <18 Years)

Who this might be right for
Fabry Disease
Amicus Therapeutics 22
Testing effectiveness (Phase 2)Looking for participantsNCT03952637
What this trial is testing

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

Who this might be right for
Lysosomal DiseasesGangliosidosisGM1
National Human Genome Research Institute (NHGRI) 54
Testing effectiveness (Phase 2)Study completedNCT00633139
What this trial is testing

Long-term Metazym Treatment of Patients With Late Infantile Metachromatic Leukodystrophy (MLD)

Who this might be right for
Late Infantile Metachromatic Leukodystrophy
Shire 13
Not applicableStudy completedNCT04997772
What this trial is testing

Digital Health Platform Customized for Patients With Gaucher Disease

Who this might be right for
Gaucher DiseasePatient Reported Outcome
Shoshana Vilk 80
Not applicableUnknownNCT03199001
What this trial is testing

Native T1 Mapping by Cardiovascular Magnetic Resonance Imaging in Rare Diseases

Who this might be right for
Fabry Disease
University College, London 400
Not applicableUnknownNCT03984149
What this trial is testing

Lipa Gene Mutation in PED-LIPIGEN (Pediatric FH Subjects)

Who this might be right for
Lysosomal Acid Lipase Deficiency
Fondazione SISA (Societa Italiana per lo Studio della Arteriosclerosi) 1,000
Not applicableAvailableNCT07762638
What this trial is testing

Post Trial Access to Lucerastat for Patients With Fabry Disease Who Participated in Study ID-069A302

Who this might be right for
Fabry Disease
Idorsia Pharmaceuticals Ltd.
Not applicableUnknownNCT05056636
What this trial is testing

Epidemiological Study of Fabry Disease Screening in Chronic Kidney Disease Patients

Who this might be right for
Fabry Disease
Chang Gung Memorial Hospital 2,000
Testing effectiveness (Phase 2)Study completedNCT00074958
What this trial is testing

Fabrazyme in Pediatric Patients With Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 16
Large-scale testing (Phase 3)Active Not RecruitingNCT04283227
What this trial is testing

OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)

Who this might be right for
Lysosomal Storage DiseasesMetachromatic Leukodystrophy
Orchard Therapeutics 6
Not applicableEnded earlyNCT02926872
What this trial is testing

Screening for Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 22
Not applicableWithdrawnNCT04462692
What this trial is testing

An Observational Study in Children With CLN2 Batten Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
REGENXBIO Inc.
Early research (Phase 1)Study completedNCT02930655
What this trial is testing

Assess the Safety and Tolerability of Lucerastat in Subjects With Fabry Disease

Who this might be right for
Fabry Disease
Idorsia Pharmaceuticals Ltd. 14
Not applicableStudy completedNCT03625882
What this trial is testing

Survey Study for Velaglucerase Alfa (VPRIV) in Japan

Who this might be right for
Gaucher Disease
Takeda 63
Not applicableAvailableNCT04316637
What this trial is testing

Early Access Program With Arimoclomol in US Patients With NPC

Who this might be right for
Niemann-Pick Disease, Type C
ZevraDenmark
Not applicableStudy completedNCT01268241
What this trial is testing

The Efficacy and Safety of Switch Between Agalsidase Beta to Agalsidase Alfa for Enzyme Replacement in Patients With Anderson-Fabry Disease

Who this might be right for
Fabry DiseaseFabry´s DiseaseAnderson-Fabry Disease
CENTOGENE GmbH Rostock 200
Testing effectiveness (Phase 2)Study completedNCT05174039
What this trial is testing

An Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of Subjects With Batten Ceroid Lipofuscinosis, Neuronal 3 (CLN3) Disease

Who this might be right for
Batten Disease
Beyond Batten Disease Foundation 6
Not applicableStudy completedNCT00351156
What this trial is testing

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Amicus Therapeutics 50
Not applicableUnknownNCT06303466
What this trial is testing

Real World Evidence Study of Danish Fabry Patients

Who this might be right for
Fabry Disease
Caroline Michaela Kistorp 115
Not applicableUnknownNCT01304875
What this trial is testing

Pulmonary Disease and Exercise Tolerance in Boys With Fabry Disease

Who this might be right for
Fabry Disease
Cedars-Sinai Medical Center 20
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