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Results for “Gaucher Disease, Type I”

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Showing 20 of 0 results

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 applicableUnknownNCT03812042
What this trial is testing

Screening of Lysosomal Storage Disorders Diseases in Minority Groups

Who this might be right for
Lysosomal Storage Diseases
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 100,000
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
Large-scale testing (Phase 3)Study completedNCT06211478
What this trial is testing

Role of Vitamin E in Gaucher Disease Patients

Who this might be right for
Gaucher Disease
Ain Shams University 40
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 applicableStudy completedNCT01136304
What this trial is testing

Validating a New Severity Score System for Adults With Type 1 Gaucher Disease (GD1)

Who this might be right for
Gaucher Disease
University Research Foundation for Lysosomal Storage Diseases, Inc. 173
Not applicableLooking for participantsNCT03291223
What this trial is testing

Gaucher Disease Outcome Survey (GOS)

Who this might be right for
Gaucher Disease
Shire 1,257
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 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 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
Large-scale testing (Phase 3)Study completedNCT00176904
What this trial is testing

Stem Cell Transplant for Inborn Errors of Metabolism

Who this might be right for
AdrenoleukodystrophyMetachromatic LeukodystrophyGloboid Cell Leukodystrophy+8 more
Masonic Cancer Center, University of Minnesota 135
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
Not applicableStudy completedNCT05908656
What this trial is testing

Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)

Who this might be right for
Gaucher Disease
Sanofi 13
Testing effectiveness (Phase 2)Study completedNCT00004488
What this trial is testing

Phase II Randomized Study of Alendronate Sodium for Osteopenia in Patients With Gaucher's Disease

Who this might be right for
Gaucher's DiseaseOsteopenia
Children's Hospital Medical Center, Cincinnati 82
Testing effectiveness (Phase 2)Ended earlyNCT04145037
What this trial is testing

Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease
AVROBIO 8
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
Large-scale testing (Phase 3)Study completedNCT00891202
What this trial is testing

Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)

Who this might be right for
Gaucher Disease, Type 1
Genzyme, a Sanofi Company 40
Testing effectiveness (Phase 2)Study completedNCT00041535
What this trial is testing

OGT 918-006: A Phase I/II Randomized, Controlled Study of OGT 918 in Patients With Neuronopathic Gaucher Disease

Who this might be right for
Gaucher Disease
National Institute of Neurological Disorders and Stroke (NINDS) 30
Not applicableUnknownNCT02000310
What this trial is testing

Molecular and Cellular Mechanisms of Lysosomal Storage Diseases

Who this might be right for
Lysosomal Storage Disorders
O & O Alpan LLC 80
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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