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Results for “Gaucher Disease, Type 1”

70 trials

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Showing 20 of 70 results

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
Testing effectiveness (Phase 2)UnknownNCT05447494
What this trial is testing

Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1Gaucher Disease, Type 3
CANbridge (Suzhou) Bio-pharma Co., Ltd. 40
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
Large-scale testing (Phase 3)Study completedNCT02770625
What this trial is testing

Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
ISU Abxis Co., Ltd. 8
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
Not applicableApproved For MarketingNCT00954460
What this trial is testing

Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 1
Shire
Post-approval studies (Phase 4)Study completedNCT02574286
What this trial is testing

Study of the Effect of Velaglucerase Alfa (VPRIV®) on Bone-related Pathology in Treatment-naïve Participants With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 21
Testing effectiveness (Phase 2)Study completedNCT00391625
What this trial is testing

Open-Label Extension Study Evaluating Long Term Safety in Patients With Type 1 Gaucher Disease Receiving DRX008A (ERT)

Who this might be right for
Gaucher Disease
Shire 10
Testing effectiveness (Phase 2)Looking for participantsNCT02583672
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 50
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
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
Early research (Phase 1)Looking for participantsNCT06517914
What this trial is testing

A First-in-Human, Single- and Multiple-Ascending Dose Study of YH35995 in Healthy Adult Male Participants

Who this might be right for
Healthy Participants
Yuhan Corporation 86
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
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)WithdrawnNCT02528617
What this trial is testing

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

Who this might be right for
Gaucher Disease Type 1Gaucher Disease Type 3
Baylor Research Institute
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
Testing effectiveness (Phase 2)Study completedNCT03950050
What this trial is testing

Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy

Who this might be right for
Gaucher Disease, Type 1
Shaare Zedek Medical Center 40
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
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 applicableStudy completedNCT05687474
What this trial is testing

Baby Detect : Genomic Newborn Screening

Who this might be right for
Congenital Adrenal HyperplasiaFamilial Hyperinsulinemic Hypoglycemia 1Phosphoglucomutase 1 Deficiency+121 more
Centre Hospitalier Universitaire de Liege 6,824
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