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Results for “Lipid Storage Disease”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Active Not RecruitingNCT05206773
What this trial is testing

Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease

Who this might be right for
Fabry Disease
Sanofi 122
Not applicableEnrolling By InvitationNCT07560956
What this trial is testing

Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)

Who this might be right for
Fabry Disease
Vastra Gotaland Region 35
Not applicableUnknownNCT04320329
What this trial is testing

Natural History of Morquio B and Late-Onset of GM1 Gangliosidosis

Who this might be right for
Morquio B DiseaseGM1 Gangliosidosis Type III
University of British Columbia 30
Not applicableNot Yet RecruitingNCT06258577
What this trial is testing

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Who this might be right for
Gaucher Disease
Chung-Hsing Wang 50
Large-scale testing (Phase 3)Ended earlyNCT01458119
What this trial is testing

Open-Label Phase 3 Long-Term Safety Study of Migalastat

Who this might be right for
Fabry Disease
Amicus Therapeutics 85
Large-scale testing (Phase 3)Study completedNCT00430625
What this trial is testing

Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
Shire 25
Not applicableLooking for participantsNCT06162338
What this trial is testing

The Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I

Who this might be right for
Gaucher Disease Type I
He Huang 3
Not applicableLooking for participantsNCT00358943
What this trial is testing

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

Who this might be right for
Gaucher DiseaseCerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+1 more
Genzyme, a Sanofi Company 12,000
Testing effectiveness (Phase 2)Active Not RecruitingNCT04127578
What this trial is testing

Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

Who this might be right for
Parkinson Disease
Prevail Therapeutics 32
Testing effectiveness (Phase 2)Study completedNCT00041535
What this trial is testing

OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease

Who this might be right for
Gaucher Disease
National Institute of Neurological Disorders and Stroke (NINDS) 30
Not applicableStudy completedNCT04469894
What this trial is testing

Health Insurance Literacy and Challenges in Accessing Health Services in Niemann-Pick

Who this might be right for
Niemann-Pick Diseases
National Niemann-Pick Disease Foundation 76
Testing effectiveness (Phase 2)Study completedNCT02112994
What this trial is testing

Safety and Efficacy Study of Sebelipase Alfa in Participants With Lysosomal Acid Lipase Deficiency

Who this might be right for
Lysosomal Acid Lipase Deficiency
Alexion Pharmaceuticals, Inc. 31
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07740512
What this trial is testing

Safety, Tolerability, Pharmacokinetics, and Preliminary Efficacy of PLX-200 in Pediatric Patients (Master Protocol)

Who this might be right for
Lysosomal Storage DisordersSandhoff DiseaseKrabbe Disease+2 more
Polaryx Therapeutics, Inc. 24
Early research (Phase 1)WithdrawnNCT01238315
What this trial is testing

Safety and Efficacy Study of HuCNS-SC in Subjects With Neuronal Ceroid Lipofuscinosis

Who this might be right for
Neuronal Ceroid Lipofuscinosis
StemCells, Inc.
Testing effectiveness (Phase 2)Study completedNCT00418847
What this trial is testing

Pharmacokinetics and Tolerability of Zavesca® (Miglustat) In Patients With Juvenile GM2 Gangliosidosis

Who this might be right for
Gangliosidoses GM2
The Hospital for Sick Children 5
Testing effectiveness (Phase 2)Looking for participantsNCT05629559
What this trial is testing

4D-310 in Adults With Fabry Disease and Cardiac Involvement

Who this might be right for
Fabry Disease
4D Molecular Therapeutics 18
Not applicableLooking for participantsNCT07545473
What this trial is testing

Retinal Hyperspectral Imaging in Neurodegenerative Diseases

Who this might be right for
DementiaNeurodegenerative DiseasesAlzheimer Disease+5 more
Center for Eye Research Australia 930
Not applicableUnknownNCT03534414
What this trial is testing

Meta-analysis of the Portfolio Dietary Pattern and Cardiometabolic Risk

Who this might be right for
Cardiovascular Risk FactorCholesterol; Lipidosis
University of Toronto 1
Not applicableStudy completedNCT04429984
What this trial is testing

Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India

Who this might be right for
Gaucher Disease
Shire 21
Testing effectiveness (Phase 2)Study completedNCT01981720
What this trial is testing

Extension Study of PRX-102 for up to 60 Months

Who this might be right for
Fabry Disease
Protalix 15
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