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Results for “Lipoidosis”

639 trials

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Showing 20 of 639 results

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
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
Testing effectiveness (Phase 2)Study completedNCT01102686
What this trial is testing

Pyrimethamine as a Treatment for Late-Onset GM2-gangliosidosis (Tay-Sachs and Sandhoff Disease)

Who this might be right for
Gangliosidoses, GM2Sandhoff DiseaseTay-Sachs Disease
The Hospital for Sick Children 20
Testing effectiveness (Phase 2)Ended earlyNCT04273269
What this trial is testing

A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis

Who this might be right for
GM1 Gangliosidosis
LYSOGENE 5
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
Early research (Phase 1)Study completedNCT02422654
What this trial is testing

Taste Evaluation of Different Liquid Formulations With Eliglustat

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 8
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT05228145
What this trial is testing

Gene Therapy Study for Children With CLN5 Batten Disease

Who this might be right for
Neuronal Ceroid Lipofuscinosis CLN5
Neurogene Inc. 6
Not applicableLooking for participantsNCT05473637
What this trial is testing

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

Who this might be right for
Cerebral Small Vessel DiseasesCadasilHTRA1-Related Autosomal Dominant Cerebral Angiopathy+5 more
National Taiwan University Hospital 500
Early research (Phase 1)WithdrawnNCT00679744
What this trial is testing

A Phase I Study of Pyrimethamine in Patients With GM2 Gangliosidosis

Who this might be right for
G(M2) GangliosideTay-Sachs Disease GangliosideSandhoff Disease Ganglioside
Exsar Corporation
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT03770572
What this trial is testing

Gene Therapy for Children With CLN3 Batten Disease

Who this might be right for
CLN3Batten Disease
Neela Therapeutics 7
Large-scale testing (Phase 3)Study completedNCT05529992
What this trial is testing

Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
Takeda 20
Testing effectiveness (Phase 2)Study completedNCT03406962
What this trial is testing

MGTA-456 in Patients With Inherited Metabolic Disorders Undergoing Hematopoietic Stem Cell Transplantation (HSCT)

Who this might be right for
Inherited Metabolic Disorders (IMD)
Magenta Therapeutics, Inc. 8
Post-approval studies (Phase 4)Study completedNCT04120506
What this trial is testing

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 15
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
Testing effectiveness (Phase 2)Ended earlyNCT03454893
What this trial is testing

Open Label, Study Of Efficacy and Safety Of AVR-RD-01 for Treatment-Naive Subjects With Classic Fabry Disease

Who this might be right for
Fabry Disease
AVROBIO 15
Not applicableUnknownNCT03542370
What this trial is testing

Umbrella Review of the DASH Dietary Pattern and Cardiometabolic Risk

Who this might be right for
Cardiovascular Risk FactorCardiovascular DiseasesCoronary Heart Disease+6 more
University of Toronto 1
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 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
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