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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableLooking for participantsNCT04613089
What this trial is testing

Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database

Who this might be right for
Neuronal Ceroid LipofuscinosisBatten DiseaseCLN1 Disease+12 more
Universitätsklinikum Hamburg-Eppendorf 500
Not applicableLooking for participantsNCT04885179
What this trial is testing

SPL Insufficiency Syndrome (SPLIS)/NPHS14: a SPLIS Observational Study and Patient Registry (International)

Who this might be right for
SphingolipidosesEnzyme Deficiency
University of California, San Francisco 120
Not applicableStudy completedNCT01355146
What this trial is testing

Home Therapy With Replagal in Fabry Disease

Who this might be right for
Fabry Disease
Shire 127
Very early researchUnknownNCT03201627
What this trial is testing

Study of Lithium Carbonate to Treat Niemann-Pick Type C1 Disease

Who this might be right for
Niemann-Pick Disease, Type C1
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 18
Not applicableStudy completedNCT01884220
What this trial is testing

Wolman/CESD Natural History Chart Review and Longitudinal Follow-Up

Who this might be right for
Wolman DiseaseCholesterol Ester Storage DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2
Children's Hospital Medical Center, Cincinnati 4
Post-approval studies (Phase 4)Study completedNCT01132690
What this trial is testing

A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Who this might be right for
Gaucher Disease
Pfizer 11
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
Testing effectiveness (Phase 2)Study completedNCT00813865
What this trial is testing

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

Who this might be right for
Gaucher DiseaseType 1 Gaucher DiseaseGaucher Disease, Type 1
Amicus Therapeutics 8
Large-scale testing (Phase 3)Study completedNCT02534844
What this trial is testing

VTS-270 to Treat Niemann-Pick Type C1 (NPC1) Disease

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 56
Testing effectiveness (Phase 2)Ended earlyNCT03746587
What this trial is testing

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Who this might be right for
Gaucher Disease, Type 1Gaucher Disease, Type 3
ZevraDenmark 39
Testing effectiveness (Phase 2)Study completedNCT00001416
What this trial is testing

Bone Response to Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 100
Early research (Phase 1)Study completedNCT02800070
What this trial is testing

Autologous Stem Cell Transplantation of Cells Engineered to Express Alpha-Galactosidase A in Patients With Fabry Disease

Who this might be right for
Fabry Disease
University Health Network, Toronto 5
Not applicableEnded earlyNCT02582294
What this trial is testing

Fabry's Disease and Pregnancy (PREFAB)

Who this might be right for
Fabry's DiseasePregnancy
Groupe Hospitalier Diaconesses Croix Saint-Simon 12
Post-approval studies (Phase 4)Study completedNCT00140621
What this trial is testing

A Safety and Efficacy Study of Fabrazyme® Replacement Therapy in Patients With Cardiac Fabry Disease

Who this might be right for
Fabry Disease
Genzyme, a Sanofi Company 6
Not applicableLooking for participantsNCT03047369
What this trial is testing

The Myelin Disorders Biorepository Project

Who this might be right for
LeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more
Children's Hospital of Philadelphia 12,000
Early research (Phase 1)Study completedNCT01730469
What this trial is testing

Safety and Pharmacokinetics of AT1001 (Migalastat HCl) in Healthy Subjects and Subjects With Impaired Renal Function

Who this might be right for
Fabry Disease
Amicus Therapeutics 32
Not applicableStudy completedNCT01136304
What this trial is testing

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

Who this might be right for
Gaucher Disease
University Research Foundation for Lysosomal Storage Diseases, Inc. 173
Not applicableLooking for participantsNCT06065852
What this trial is testing

National Registry of Rare Kidney Diseases

Who this might be right for
Adenine Phosphoribosyltransferase DeficiencyAH AmyloidosisAHL Amyloidosis+81 more
UK Kidney Association 35,000
Not applicableLooking for participantsNCT01581424
What this trial is testing

Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal Disease Study

Who this might be right for
Fabry Disease
University of Minnesota 50
Not applicableNo Longer AvailableNCT02376751
What this trial is testing

An Expanded Access Protocol for Sebelipase Alfa for Patients With Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc.
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