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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT01793168
What this trial is testing

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

Who this might be right for
Rare DisordersUndiagnosed DisordersDisorders of Unknown Prevalence+340 more
Sanford Health 20,000
Testing effectiveness (Phase 2)Study completedNCT02678689
What this trial is testing

A Safety, Tolerability, and Efficacy Study of BMN 190 in Pediatric Patients < 18 Years of Age With CLN2 Disease

Who this might be right for
Jansky-Bielschowsky DiseaseBatten DiseaseLate-Infantile Neuronal Ceroid Lipofuscinosis Type 2+2 more
BioMarin Pharmaceutical 14
Testing effectiveness (Phase 2)Active Not RecruitingNCT05739643
What this trial is testing

Gene Transfer Clinical Trial for Infantile and Late Infantile Krabbe Disease Treated Previously With HSCT

Who this might be right for
Krabbe Disease
Forge Biologics, Inc 9
Large-scale testing (Phase 3)Study completedNCT01757184
What this trial is testing

Acid Lipase Replacement Investigating Safety and Efficacy (ARISE) in Participants With Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 66
Testing effectiveness (Phase 2)Study completedNCT03471143
What this trial is testing

Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C

Who this might be right for
Niemann-Pick Disease, Type C
Washington University School of Medicine 4
Not applicableStudy completedNCT00001774
What this trial is testing

Vasodilation in Patients With Fabry's Disease

Who this might be right for
Cerebrovascular AccidentFabry DiseaseHealthy
National Institute of Neurological Disorders and Stroke (NINDS) 48
Early research (Phase 1)Study completedNCT00001410
What this trial is testing

PEG-Glucocerebrosidase for the Treatment of Gaucher Disease

Who this might be right for
Gaucher's Disease
National Institute of Mental Health (NIMH) 18
Large-scale testing (Phase 3)Study completedNCT01371825
What this trial is testing

Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase DeficiencyWolman Disease
Alexion Pharmaceuticals, Inc. 9
Large-scale testing (Phase 3)Study completedNCT02612129
What this trial is testing

Arimoclomol Prospective Study in Participants Diagnosed With Niemann-Pick Disease Type C

Who this might be right for
Niemann-Pick Disease, Type C
ZevraDenmark 50
Not applicableWithdrawnNCT00795197
What this trial is testing

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

Who this might be right for
Gaucher Disease, Type 1
Genzyme, a Sanofi Company
Not applicableEnded earlyNCT01963650
What this trial is testing

Natural History Study of Children With Metachromatic Leukodystrophy

Who this might be right for
Lipid Metabolism DisordersMetachromatic Leukodystrophy (MLD)Nervous System Diseases+11 more
Shire 1
Not applicableActive Not RecruitingNCT06095713
What this trial is testing

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

Who this might be right for
Fabry Disease
Universität Münster 60
Not applicableActive Not RecruitingNCT01911091
What this trial is testing

Identification of Novel Skeletal Muscle-derived Factors That Promote Lipid Oxidation (Columbus)

Who this might be right for
ObesityDisorder of Lipid Storage and MetabolismLipid Metabolism Disorders+1 more
AdventHealth Translational Research Institute 56
Not applicableActive Not RecruitingNCT03305250
What this trial is testing

Arrhythmia Burden, Risk of Sudden Cardiac Death and Stroke in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University Hospital Birmingham NHS Foundation Trust 169
Not applicableStudy completedNCT04652713
What this trial is testing

Breakfast for Young Women

Who this might be right for
ObesityWolman DiseaseNutrition
University of Aarhus 30
Not applicableLooking for participantsNCT06806813
What this trial is testing

Italian Anderson Fabry Disease Cardiovascular Registry

Who this might be right for
Fabry Disease
IRCCS Azienda Ospedaliero-Universitaria di Bologna 800
Large-scale testing (Phase 3)Study completedNCT02536755
What this trial is testing

Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 31
Not applicableEnrolling By InvitationNCT00001215
What this trial is testing

Genetic Studies of Lysosomal Storage Disorders

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseParkinson Disease
National Human Genome Research Institute (NHGRI) 1,000
Testing effectiveness (Phase 2)Study completedNCT05443685
What this trial is testing

ADX-629 Therapy for Sjogren-Larsson Syndrome

Who this might be right for
Sjogren-Larsson Syndrome
University of Nebraska 8
Testing effectiveness (Phase 2)UnknownNCT05768035
What this trial is testing

Safety and Efficacy of SMART101 in Adult Patients With Hematological Malignancies After Haploidentical HSCT With Post-transplant Cyclophosphamide

Who this might be right for
Hematological Malignancies
Smart Immune SAS 40
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