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Results for “Gaucher Disease, Type 1”

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Showing 20 of 0 results

Testing effectiveness (Phase 2)Study completedNCT00001416
What this trial is testing

Bone Response to Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 100
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 completedNCT00478647
What this trial is testing

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

Who this might be right for
Gaucher Disease
Shire 40
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 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 participantsNCT06162338
What this trial is testing

The Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Disease Type I

Who this might be right for
Gaucher Disease Type I
He Huang 3
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
Early research (Phase 1)Study completedNCT06143904
What this trial is testing

Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants

Who this might be right for
Gaucher's Disease Type I
Sanofi 10
Testing effectiveness (Phase 2)Active Not RecruitingNCT06545136
What this trial is testing

Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2)

Who this might be right for
Gaucher Disease, Type 1
Spur Therapeutics 75
Testing effectiveness (Phase 2)Study completedNCT00358150
What this trial is testing

The Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

Who this might be right for
Gaucher Disease, Type 1Cerebroside Lipidosis SyndromeGlucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 26
Early research (Phase 1)Ended earlyNCT00875160
What this trial is testing

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

Who this might be right for
Type 1 Gaucher Disease
Amicus Therapeutics 1
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
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
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
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
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
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
Testing effectiveness (Phase 2)Active Not RecruitingNCT02843035
What this trial is testing

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension

Who this might be right for
Gaucher Disease Type 1Gaucher Disease Type 3
Genzyme, a Sanofi Company 12
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