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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Ended earlyNCT03879655
What this trial is testing

Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1

Who this might be right for
Niemann-Pick Disease, Type C
Mandos LLC 2
Large-scale testing (Phase 3)Study completedNCT02795676
What this trial is testing

Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function

Who this might be right for
Fabry Disease
Protalix 78
Large-scale testing (Phase 3)Study completedNCT00478647
What this trial is testing

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

Who this might be right for
Gaucher Disease
Shire 40
Not applicableEnded earlyNCT02345421
What this trial is testing

Identify and Characterize LAL-D Patients in High-risk Populations

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 640
Not applicableUnknownNCT00871611
What this trial is testing

Viennese Prevalence Study of Anderson-Fabry Disease

Who this might be right for
Fabry DiseaseLeft Ventricular Hypertrophy
Medical University of Vienna 4,000
Testing effectiveness (Phase 2)Study completedNCT00075244
What this trial is testing

Alternative Dosing and Regimen of Replagal to Treat Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 25
Not applicableUnknownNCT00005900
What this trial is testing

Study of Pulmonary Complications in Pediatric Patients With Storage Disorders Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Who this might be right for
I Cell DiseaseFucosidosisGloboid Cell Leukodystrophy+9 more
Fairview University Medical Center 10
Not applicableLooking for participantsNCT04528355
What this trial is testing

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more
Paul Szabolcs 50
Testing effectiveness (Phase 2)Active Not RecruitingNCT04798235
What this trial is testing

First-in-Human Study of TSHA-101 Gene Therapy for Treatment of Infantile Onset GM2 Gangliosidosis

Who this might be right for
Infantile GM2 Gangliosidosis (Disorder)
Dr. Anupam Sehgal 3
Large-scale testing (Phase 3)Study completedNCT04974749
What this trial is testing

REPLAGAL® in Treatment-naive Chinese Participants With Fabry Disease

Who this might be right for
Fabry Disease
Takeda 20
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
Not applicableLooking for participantsNCT06758648
What this trial is testing

Characterizing the Retinal Microvasculature in Patients with Fabry Disease: a Prospective Observational Study

Who this might be right for
Fabry DiseaseEndothelial DysfunctionMicrovasculature
Technical University of Munich 63
Early research (Phase 1)Study completedNCT05324943
What this trial is testing

A Gene Therapy Study in Patients With Gaucher Disease Type 1

Who this might be right for
Gaucher Disease, Type 1
Spur Therapeutics 10
Testing effectiveness (Phase 2)Study completedNCT01043640
What this trial is testing

Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

Who this might be right for
MucopolysaccharidosisHurler SyndromeHunter Syndrome+10 more
Masonic Cancer Center, University of Minnesota 46
Not applicableStudy completedNCT03883750
What this trial is testing

Induced Pluripotent Stem Cells for Niemann Pick Disease

Who this might be right for
Niemann-Pick Diseases
CENTOGENE GmbH Rostock 40
Not applicableStudy completedNCT04804566
What this trial is testing

Understanding Fabry Disease Therapy Choices Through the Eyes of the Patients

Who this might be right for
Fabry Disease
Amicus Therapeutics 44
Not applicableStudy completedNCT01528917
What this trial is testing

An Observational Study of Patients With Lysosomal Acid Lipase Deficiency/Cholesteryl Ester Storage Disease Phenotype

Who this might be right for
Cholesterol Ester Storage Disease(CESD)Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 49
Not applicableStudy completedNCT01466816
What this trial is testing

Lipids and Insulin Sensitivity

Who this might be right for
ObesityInsulin Resistance
Maastricht University Medical Center 10
Not applicableWithdrawnNCT01306604
What this trial is testing

Biomarker for Niemann Pick Type C Disease (BioNPC)

Who this might be right for
Niemann-Pick DiseaseNiemann-Pick Disease, Type C
CENTOGENE GmbH Rostock
Not applicableUnknownNCT03596398
What this trial is testing

Epidemiological Study of Fabry Disease in Taiwan Young Stroke Patients

Who this might be right for
Fabry Disease
Chiayi Christian Hospital 1,000
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