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Results for “Type I Gaucher Disease”

71 trials

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Showing 20 of 71 results

Not applicableStudy completedNCT00351156
What this trial is testing

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Amicus Therapeutics 50
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
Early research (Phase 1)Looking for participantsNCT03721068
What this trial is testing

Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma

Who this might be right for
NeuroblastomaOsteosarcoma
UNC Lineberger Comprehensive Cancer Center 18
Not applicableUnknownNCT04055831
What this trial is testing

Immune Biomarkers Related to Bone Pathology in Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease Type 1
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 40
Not applicableLooking for participantsNCT05253560
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)Temporarily pausedNCT01463215
What this trial is testing

Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease

Who this might be right for
Type I Gaucher Disease
Exsar Corporation 20
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
Not applicableStudy completedNCT02067247
What this trial is testing

Comparison of BMD Measurement by DEXA to BeamMed Speed-of-Sound Measurement at Forearm in Patients With Gaucher Disease

Who this might be right for
Gaucher Disease Type 1
Shaare Zedek Medical Center 100
Not applicableLooking for participantsNCT02437396
What this trial is testing

Oxidative Stress and Inflammatory Biomarkers in Gaucher Disease

Who this might be right for
Gaucher Disease Type IOxidative StressInflammation
University of Minnesota 34
Not applicableWithdrawnNCT00795197
What this trial is testing

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

Who this might be right for
Gaucher Disease, Type 1
Genzyme, a Sanofi Company
Post-approval studies (Phase 4)WithdrawnNCT03021941
What this trial is testing

Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease

Who this might be right for
Type 1 Gaucher Disease
Pfizer
Early research (Phase 1)Study completedNCT05324943
What this trial is testing

A Gene Therapy Study in Patients With Gaucher Disease Type 1

Who this might be right for
Gaucher Disease, Type 1
Spur Therapeutics 10
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
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
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
Very early researchActive Not RecruitingNCT06528080
What this trial is testing

A Clinical Study for the Treatment of Pediatric and Adolescent Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
Shanghai Jiao Tong University School of Medicine 3
Not applicableLooking for participantsNCT04528355
What this trial is testing

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+2 more
Paul Szabolcs 50
Testing effectiveness (Phase 2)Looking for participantsNCT01962415
What this trial is testing

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who this might be right for
Primary Immunodeficiency (PID)Congenital Bone Marrow Failure SyndromesInherited Metabolic Disorders (IMD)+4 more
Paul Szabolcs 100
Post-approval studies (Phase 4)Study completedNCT03702361
What this trial is testing

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease

Who this might be right for
Primary Disease
Shaare Zedek Medical Center 15
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
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