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Results for “Gaucher”

173 trials

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Showing 20 of 173 results

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
Early research (Phase 1)Study completedNCT00004294
What this trial is testing

Phase I Study of Retrovirally Mediated Transfer of the Human Glucocerebrosidase Gene Into Peripheral Blood Stem Cells for Autologous Transplantation in Patients With Type I Gaucher Disease

Who this might be right for
Gaucher's Disease
National Center for Research Resources (NCRR) 15
Large-scale testing (Phase 3)Study completedNCT01614574
What this trial is testing

Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 6
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)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
Large-scale testing (Phase 3)Study completedNCT01422187
What this trial is testing

Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

Who this might be right for
Gaucher Disease
Pfizer 19
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 applicableActive Not RecruitingNCT06488261
What this trial is testing

Long Term Follow-Up for Safety of AVR-RD-02

Who this might be right for
Gaucher Disease
John Bernat 2
Not applicableStudy completedNCT02785744
What this trial is testing

Genzyme Osteopenia/Osteoporosis Study

Who this might be right for
Gaucher Disease
NYU Langone Health 76
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 completedNCT04002830
What this trial is testing

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 3
Ari Zimran 14
Not applicableLooking for participantsNCT05619900
What this trial is testing

Registry of Patients Diagnosed With Lysosomal Storage Diseases

Who this might be right for
Mucopolysaccharidosis IMucopolysaccharidosis IIMucopolysaccharidosis IV A+5 more
University of California, San Francisco 250
Not applicableUnknownNCT02120235
What this trial is testing

Investigating Lysosomal Storage Diseases in Minority Groups

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseFabry Disease+2 more
O & O Alpan LLC 20,000
Post-approval studies (Phase 4)Study completedNCT04656600
What this trial is testing

Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ

Who this might be right for
Gaucher's Disease
Sanofi 12
Testing effectiveness (Phase 2)Looking for participantsNCT06818838
What this trial is testing

A Clinical Study Evaluating LY-M001 Injection in the Treatment of Adult Patients With Type I Gaucher Disease

Who this might be right for
Gaucher Disease Type 1
Lingyi Biotech Co., Ltd. 12
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
Not applicableStudy completedNCT01439607
What this trial is testing

Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease

Who this might be right for
Gaucher Disease
Assistance Publique - Hôpitaux de Paris 30
Not applicableLooking for participantsNCT06573723
What this trial is testing

Institutional Registry of Rare Diseases

Who this might be right for
Rare DiseasesAmyloidosisSarcoidosis+23 more
Hospital Italiano de Buenos Aires 380
Testing effectiveness (Phase 2)Active Not RecruitingNCT04411654
What this trial is testing

Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

Who this might be right for
Gaucher Disease, Type 2
Prevail Therapeutics 7
Very early researchLooking for participantsNCT06272149
What this trial is testing

An Exploratory Clinical Trial of VGN-R08b in Patients With Type II Gaucher Disease

Who this might be right for
Type II Gaucher Disease
Xinhua Hospital, Shanghai Jiao Tong University School of Medicine 6
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