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Results for “Lipid Storage Disease”

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Enrolling by Invitation

Showing 20 of 0 results

Early research (Phase 1)Study completedNCT01586455
What this trial is testing

Human Placental-Derived Stem Cell Transplantation

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis VIAdrenoleukodystrophy+13 more
New York Medical College 43
Not applicableStudy completedNCT03678324
What this trial is testing

Pre-Clinical White Matter Changes and Associated Connectivity Effects in Fabry Disease

Who this might be right for
Fabry Disease
University of Utah 40
Testing effectiveness (Phase 2)Ended earlyNCT00837824
What this trial is testing

Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme

Who this might be right for
Fabry DiseaseChronic Kidney Disease, Stage IV (Severe)
Genzyme, a Sanofi Company 20
Not applicableEnrolling By InvitationNCT06627543
What this trial is testing

Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3

Who this might be right for
Gaucher Disease Type 3
Noha Osman 44
Testing effectiveness (Phase 2)Active Not RecruitingNCT04519749
What this trial is testing

An Open-label, Phase 1/2 Trial of Gene Therapy 4D-310 in Adults With Fabry Disease

Who this might be right for
Fabry Disease
4D Molecular Therapeutics 18
Not applicableUnknownNCT01274208
What this trial is testing

Examining the Immune Response in Patients With Gaucher Disease and Hepatitis C

Who this might be right for
Gaucher DiseaseHepatitis C
Shaare Zedek Medical Center 80
Not applicableStudy completedNCT00055016
What this trial is testing

Registry of Fabry Disease - A Multicenter Observational Study

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 100
Very early researchLooking for participantsNCT06207552
What this trial is testing

Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease

Who this might be right for
Fabry Disease
Children's Hospital of Fudan University 6
Testing effectiveness (Phase 2)Study completedNCT03392987
What this trial is testing

A Safety and Efficacy Study of Cryopreserved OTL-200 for Treatment of Metachromatic Leukodystrophy (MLD)

Who this might be right for
Lysosomal Storage DiseaseMetachromatic Leukodystrophy
Orchard Therapeutics 10
Large-scale testing (Phase 3)Active Not RecruitingNCT07082543
What this trial is testing

Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis

Who this might be right for
Gangliosidoses, GM2Gangliosidosis, GM1
Azafaros B.V. 75
Early research (Phase 1)Study completedNCT01653444
What this trial is testing

Evaluate the Safety and Exploratory Efficacy of GC1119

Who this might be right for
Fabry Disease
Green Cross Corporation 8
Not applicableStudy completedNCT00030134
What this trial is testing

Data Collection in Women With Fabry Disease

Who this might be right for
Fabry Disease
National Institute of Neurological Disorders and Stroke (NINDS) 60
Large-scale testing (Phase 3)Study completedNCT03425539
What this trial is testing

Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease

Who this might be right for
Fabry Disease
Idorsia Pharmaceuticals Ltd. 118
Early research (Phase 1)Not Yet RecruitingNCT07479953
What this trial is testing

Prenatal Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis Clinical Trial

Who this might be right for
GM1 GangliosidosesGM1 Gangliosidosis, Type IGM1 Gangliosidosis, Type 2
Tippi Mackenzie 5
Not applicableUnknownNCT05679076
What this trial is testing

A Qualitative Study on the Pain and Quality of Life of Patients With Fabry Disease

Who this might be right for
Fabry Disease
Yonsei University 10
Not applicableStudy completedNCT00465062
What this trial is testing

Evaluate and Characterize the Effect of Pharmacological Chemicals on Blood From Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
National Institute of Neurological Disorders and Stroke (NINDS) 50
Large-scale testing (Phase 3)Active Not RecruitingNCT05222906
What this trial is testing

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Who this might be right for
Gaucher's Disease Type III
Sanofi 43
Not applicableUnknownNCT05536388
What this trial is testing

Drug Discovery for Parkinson's With Mutations in the GBA Gene

Who this might be right for
Parkinson DiseaseGaucher DiseaseHealthy+1 more
New York Stem Cell Foundation Research Institute 60
Testing effectiveness (Phase 2)Study completedNCT01303146
What this trial is testing

Efficacy METAZYM for the Treatment Metachromatic Leukodystrophy Treated With Hematopoietic Stem Cell Transplantation

Who this might be right for
Metachromatic Leukodystrophy
Assistance Publique - Hôpitaux de Paris 1
Not applicableStudy completedNCT04808297
What this trial is testing

Trehalose in Subjects With Neuronal Ceroid Lipofuscinoses

Who this might be right for
Neuronal Ceroid-Lipofuscinoses
IRCCS Fondazione Stella Maris 8
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