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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Not applicableStudy completedNCT02053896
What this trial is testing

A Switch-Over Study of the Safety and Efficacy of ISU302 in Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
ISU Abxis Co., Ltd. 5
Not applicableLooking for participantsNCT07382128
What this trial is testing

Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes

Who this might be right for
HCM - Hypertrophic CardiomyopathyAnderson Fabry DiseaseCardiac Magnetic Resonance Imaging+1 more
IRCCS Azienda Ospedaliero-Universitaria di Bologna 250
Not applicableUnknownNCT04055831
What this trial is testing

Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease Type 1
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 40
Testing effectiveness (Phase 2)UnknownNCT06114329
What this trial is testing

Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease

Who this might be right for
Fabry Disease
AceLink Therapeutics, Inc. 16
Not applicableStudy completedNCT04041102
What this trial is testing

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

Who this might be right for
GM1 Gangliosidosis
University of Pennsylvania 31
Not applicableStudy completedNCT00343577
What this trial is testing

Antiproteinuric Agents and Fabry Disease

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 12
Large-scale testing (Phase 3)Looking for participantsNCT07223944
What this trial is testing

A Gaucher Disease Gene Therapy Trial With FLT201

Who this might be right for
Gaucher Disease Type 1
Spur Therapeutics 45
Not applicableLooking for participantsNCT06610019
What this trial is testing

Cardiovascular Multimodality Imaging Study

Who this might be right for
Non-ischemic CardiomyopathyCardiomyopathiesHypertrophic Cardiomyopathy+7 more
Montefiore Medical Center 10,000
Not applicableStudy completedNCT05641103
What this trial is testing

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase DeficiencyASMD+2 more
Fundación Española de Hematología y Hemoterapía 122
Not applicableWithdrawnNCT02778295
What this trial is testing

Biomarker for Patients With Fabry Disease (BioFabry)

Who this might be right for
AngiokeratomasChronic Kidney DiseaseOcular Abnormalities+1 more
CENTOGENE GmbH Rostock
Testing effectiveness (Phase 2)Study completedNCT01307098
What this trial is testing

Safety, Tolerability and Pharmacokinetics of SBC-102 (Sebelipase Alfa) in Adult Participants With Lysosomal Acid Lipase Deficiency

Who this might be right for
Cholesterol Ester Storage Disease(CESD)Lysosomal Acid Lipase DeficiencyLAL-Deficiency
Alexion Pharmaceuticals, Inc. 9
Not applicableLooking for participantsNCT00668187
What this trial is testing

A Natural History Study of the Gangliosidoses

Who this might be right for
Tay-Sachs DiseaseSandhoff DiseaseLate Onset Tay-Sachs Disease+2 more
University of Minnesota 52
Testing effectiveness (Phase 2)Active Not RecruitingNCT04411654
What this trial is testing

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

Who this might be right for
Gaucher Disease, Type 2
Prevail Therapeutics 7
Not applicableStudy completedNCT01999257
What this trial is testing

Efficacy Study of an Online Educational Module Before Carrier Genetic Screening in Persons of Ashkenazi Jewish Descent.

Who this might be right for
Tay Sachs DiseaseCanavan DiseaseFamilial Dysautonomia
McGill University Health Centre/Research Institute of the McGill University Health Centre 60
Large-scale testing (Phase 3)Study completedNCT01411228
What this trial is testing

Extension Study of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Who this might be right for
Gaucher Disease
Pfizer 15
Testing effectiveness (Phase 2)Ended earlyNCT01372228
What this trial is testing

Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders

Who this might be right for
Hurler Syndrome (MPS I)Hurler-Scheie SyndromeHunter Syndrome (MPS II)+9 more
Talaris Therapeutics Inc. 3
Testing effectiveness (Phase 2)Study completedNCT01304277
What this trial is testing

This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.

Who this might be right for
Fabry Disease
Shire 17
Early research (Phase 1)Study completedNCT00004294
What this trial is testing

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

Who this might be right for
Gaucher's Disease
National Center for Research Resources (NCRR) 15
Not applicableUnknownNCT01344096
What this trial is testing

Thrombocytopathy in Gaucher Disease Patients

Who this might be right for
Gaucher DiseaseThrombocytopathy
Rabin Medical Center 70
Not applicableStudy completedNCT00446862
What this trial is testing

The Fabrazyme® and Arbs and ACE Inhibitor Treatment (FAACET) Study

Who this might be right for
Fabry DiseaseProteinuria
University of Alabama at Birmingham 36
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