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Results for “Glucosylceramide Beta-Glucosidase Deficiency Disease”

Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT06211478
What this trial is testing

Role of Vitamin E in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Ain Shams University 40
Not applicableLooking for participantsNCT05992532
What this trial is testing

GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase Deficiency
Fundación Española de Hematología y Hemoterapía 210
Testing effectiveness (Phase 2)UnknownNCT01951989
What this trial is testing

Intra-monocyte Imiglucerase Kinetics in Gaucher Disease

Who this might be right for
Gaucher Disease
University Hospital, Clermont-Ferrand 60
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
Not applicableUnknownNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
Not applicableLooking for participantsNCT03240653
What this trial is testing

Gaucherite - A Study to Stratify Gaucher Disease

Who this might be right for
Gaucher Disease, Type IGaucher Disease, Type III
Cambridge University Hospitals NHS Foundation Trust 250
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
Large-scale testing (Phase 3)Looking for participantsNCT07223944
What this trial is testing

A Gaucher Disease Gene Therapy Trial With FLT201

Who this might be right for
Gaucher Disease Type 1
Spur Therapeutics 45
Large-scale testing (Phase 3)Study completedNCT01074944
What this trial is testing

Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 170
Testing effectiveness (Phase 2)Study completedNCT00433147
What this trial is testing

AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy

Who this might be right for
Gaucher Disease, Type 1Type 1 Gaucher DiseaseGaucher Disease
Amicus Therapeutics 30
Not applicableNot Yet RecruitingNCT05669729
What this trial is testing

A Survey to Assess Participants', Caregivers', and Nurses' Use and Understanding of Educational Material on Velaglucerase Alfa (VPRIV) Home Infusion

Who this might be right for
Gaucher Disease
Takeda 60
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
Not applicableStudy completedNCT05908656
What this trial is testing

Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)

Who this might be right for
Gaucher Disease
Sanofi 13
Not applicableWithdrawnNCT01331642
What this trial is testing

Biomarker for Gaucher Disease: BioGaucher (BioGaucher)

Who this might be right for
SplenomegalyHepatomegaly
CENTOGENE GmbH Rostock
Early research (Phase 1)Study completedNCT01747980
What this trial is testing

Safety and Pharmacokinetics of Oral PRX-112 in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Protalix 16
Post-approval studies (Phase 4)Study completedNCT04718779
What this trial is testing

Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

Who this might be right for
Gaucher Disease
Takeda 4
Early research (Phase 1)Study completedNCT06193304
What this trial is testing

Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth

Who this might be right for
Gaucher's Disease
Sanofi 6
Not applicableEnded earlyNCT04836377
What this trial is testing

A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

Who this might be right for
Type 1 Gaucher Disease
AVROBIO 4
Early research (Phase 1)Study completedNCT02536937
What this trial is testing

The Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate

Who this might be right for
Gaucher Disease
Sanofi 32
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
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