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Results for “Gaucher”

172 trials

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Showing 20 of 172 results

Early research (Phase 1)Study completedNCT00744692
What this trial is testing

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Who this might be right for
Non Malignant DisordersImmunodeficienciesCongenital Marrow Failures+5 more
Duke University 22
Large-scale testing (Phase 3)Study completedNCT00891202
What this trial is testing

Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

Who this might be right for
Gaucher Disease, Type 1
Genzyme, a Sanofi Company 40
Not applicableUnknownNCT03811496
What this trial is testing

Biomarker Analysis for GBA Associated Parkinson's Disease

Who this might be right for
Parkinson DiseaseGaucher Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 100
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 applicableEnrolling By InvitationNCT05368038
What this trial is testing

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program

Who this might be right for
Acid Sphingomyelinase DeficiencyCeroid Lipofuscinosis, Neuronal, 2Cerebrotendinous Xanthomatosis+11 more
Albert Einstein College of Medicine 100,000
Early research (Phase 1)Study completedNCT01427517
What this trial is testing

Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease

Who this might be right for
Parkinson's DiseaseGaucher's Disease
University of Minnesota 9
Large-scale testing (Phase 3)Study completedNCT00635427
What this trial is testing

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
Shire 95
Not applicableStudy completedNCT03333447
What this trial is testing

Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa)

Who this might be right for
Gaucher Disease, Type 1MRI
CEN Biotech 20
Large-scale testing (Phase 3)Study completedNCT00553631
What this trial is testing

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
Shire 34
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 completedNCT01358474
What this trial is testing

Detecting Dopaminergic Deficits in Individuals At-risk for Parkinsonism

Who this might be right for
Parkinson DiseaseGaucher DiseaseIdiopathic Rapid Eye Movement Sleep Disorder
University of Minnesota 56
Not applicableUnknownNCT05253560
What this trial is testing

Prodromal Parkinsonian Features in GBA1 Mutation Carriers

Who this might be right for
Gaucher Disease, Type 1Healthy
Shaare Zedek Medical Center 600
Testing effectiveness (Phase 2)Study completedNCT02107846
What this trial is testing

An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112

Who this might be right for
Gaucher Disease
Protalix 10
Testing effectiveness (Phase 2)Study completedNCT02583672
What this trial is testing

Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1)

Who this might be right for
Gaucher Disease Type 1
University of Minnesota 33
Post-approval studies (Phase 4)Study completedNCT00364858
What this trial is testing

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1Cerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 95
Not applicableWithdrawnNCT04189601
What this trial is testing

Complement Activation in the Lysosomal Storage Disorders

Who this might be right for
Fabry DiseaseGaucher DiseaseNiemann-Pick Disease, Type C+1 more
Melbourne Health
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
Not applicableUnknownNCT03812042
What this trial is testing

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

Who this might be right for
Lysosomal Storage Diseases
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 100,000
Large-scale testing (Phase 3)Study completedNCT02536755
What this trial is testing

Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 31
Not applicableStudy completedNCT03519646
What this trial is testing

Eliglustat on Gaucher Disease Type IIIB

Who this might be right for
Gaucher Disease, Type III
National Taiwan University Hospital 4
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