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Results for “Gaucher Disease”

175 trials

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Showing 20 of 175 results

Large-scale testing (Phase 3)Study completedNCT03485677
What this trial is testing

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Who this might be right for
Gaucher's Disease Type IGaucher's Disease Type III
Sanofi 57
Not applicableEnded earlyNCT03896607
What this trial is testing

Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography

Who this might be right for
Gaucher DiseaseLiver FibrosisSpleen; Fibrosis
HaEmek Medical Center, Israel 18
Testing effectiveness (Phase 2)Study completedNCT01685216
What this trial is testing

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 3
Shire 7
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
Testing effectiveness (Phase 2)Study completedNCT00813865
What this trial is testing

A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients

Who this might be right for
Gaucher DiseaseType 1 Gaucher DiseaseGaucher Disease, Type 1
Amicus Therapeutics 8
Post-approval studies (Phase 4)Study completedNCT04120506
What this trial is testing

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 15
Not applicableLooking for participantsNCT05843552
What this trial is testing

Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease

Who this might be right for
Gaucher Disease
University of Minnesota 30
Not applicableStudy completedNCT04997772
What this trial is testing

Digital Health Platform Customized for Patients With Gaucher Disease

Who this might be right for
Gaucher DiseasePatient Reported Outcome
Shoshana Vilk 80
Not applicableWithdrawnNCT01331642
What this trial is testing

Biomarker for Gaucher Disease: BioGaucher (BioGaucher)

Who this might be right for
SplenomegalyHepatomegaly
CENTOGENE GmbH Rostock
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
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
Early research (Phase 1)Study completedNCT02536911
What this trial is testing

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

Who this might be right for
Gaucher Disease
Genzyme, a Sanofi Company 24
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
Large-scale testing (Phase 3)WithdrawnNCT01161914
What this trial is testing

The Safety and Efficacy Study of ISU302 in Patient With Type I Gaucher Disease

Who this might be right for
Gaucher Disease
ISU Abxis Co., Ltd.
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 applicableStudy completedNCT05641103
What this trial is testing

PREDIGA 2: Spanish Acronym of "Educational and Diagnostic Project for Gaucher and ASMD"

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase DeficiencyASMD+2 more
Fundación Española de Hematología y Hemoterapía 122
Testing effectiveness (Phase 2)UnknownNCT04637503
What this trial is testing

4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma

Who this might be right for
Neuroblastoma
Shenzhen Geno-Immune Medical Institute 100
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 applicableNo Longer AvailableNCT00962260
What this trial is testing

Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Pfizer
Not applicableStudy completedNCT02416661
What this trial is testing

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

Who this might be right for
Lysosomal Storage DiseasesGaucher DiseaseSphingolipidoses
CENTOGENE GmbH Rostock 299
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