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Results for “Type II Gaucher Disease”

20 trials

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Showing 20 of 20 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
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 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
Not applicableStudy completedNCT00244582
What this trial is testing

Convection-Enhanced Delivery of Glucocerebrosidase to Treat Type 2 Gaucher Disease

Who this might be right for
Type 2 Gaucher Disease
National Institute of Neurological Disorders and Stroke (NINDS) 1
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
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
Testing effectiveness (Phase 2)Not Yet RecruitingNCT07603050
What this trial is testing

A Phase I/II Clinical Study to Evaluate the Safety and Efficacy of VGN-R08b in Patients With Type III Gaucher's Disease

Who this might be right for
Gaucher Disease Type 3
Shanghai Vitalgen BioPharma Co., Ltd. 12
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
Not applicableUnknownNCT06539169
What this trial is testing

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Who this might be right for
Alpha-ThalassemiaBeta-ThalassemiaAmyloidosis+14 more
xCures 1,000
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 applicableUnknownNCT02605603
What this trial is testing

SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease

Who this might be right for
Gaucher Disease
Lysosomal and Rare Disorders Research and Treatment Center, Inc. 30
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
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
Early research (Phase 1)Study completedNCT00001410
What this trial is testing

PEG-Glucocerebrosidase for the Treatment of Gaucher Disease

Who this might be right for
Gaucher's Disease
National Institute of Mental Health (NIMH) 18
Not applicableUnknownNCT04758130
What this trial is testing

Getting Global Rare Disease Insights Through Technology Study

Who this might be right for
Metabolic DiseaseFabry DiseaseGaucher Disease+2 more
M.A.G.I.C. Clinic LTD 150
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
Not applicableStudy completedNCT04470713
What this trial is testing

Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2

Who this might be right for
GM1 GangliosidosisGM2 GangliosidosisGaucher Disease, Type 2+3 more
Idorsia Pharmaceuticals Ltd. 226
Testing effectiveness (Phase 2)Not Yet RecruitingNCT04637503
What this trial is testing

4SCAR-T Therapy Targeting GD2, PSMA and CD276 for Treating Neuroblastoma

Who this might be right for
Neuroblastoma
Shenzhen Geno-Immune Medical Institute 100
Early research (Phase 1)Looking for participantsNCT04532047
What this trial is testing

PEARL (PrEnAtal Enzyme Replacement Therapy for Lysosomal Storage Disorders)

Who this might be right for
MPS IMPS IIMPS IVA+6 more
University of California, San Francisco 10
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