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Results for “Neuronopathic Gaucher Disease”

31 trials

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Showing 20 of 31 results

Testing effectiveness (Phase 2)Ended earlyNCT03746587
What this trial is testing

Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3

Who this might be right for
Gaucher Disease, Type 1Gaucher Disease, Type 3
ZevraDenmark 39
Not applicableStudy completedNCT00001289
What this trial is testing

Effects of Enzyme Replacement in Gaucher's Disease

Who this might be right for
Gaucher's Disease
National Institute of Neurological Disorders and Stroke (NINDS) 70
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
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 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
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
Not applicableActive Not RecruitingNCT07285369
What this trial is testing

High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)

Who this might be right for
Gaucher Disease, Type 3
Agyany Pharma LTD 12
Post-approval studies (Phase 4)Study completedNCT00365131
What this trial is testing

Study of the Efficacy of Cerezyme in Testing Skeletal Disease in Patients With Type I Gaucher Disease.

Who this might be right for
Gaucher Disease Type ICerebroside Lipidosis SyndromeClucocerebrosidase Deficiency Disease+2 more
Genzyme, a Sanofi Company 40
Not applicableLooking for participantsNCT05586243
What this trial is testing

MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)

Who this might be right for
Gaucher Disease, Type 3
University of Minnesota 5
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)Active Not RecruitingNCT00911560
What this trial is testing

Bivalent Vaccine With Escalating Doses of the Immunological Adjuvant OPT-821, in Combination With Oral β-glucan for High-Risk Neuroblastoma

Who this might be right for
Neuroblastoma
Memorial Sloan Kettering Cancer Center 374
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
Testing effectiveness (Phase 2)Study completedNCT01685216
What this trial is testing

Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 3
Shire 7
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 applicableStudy completedNCT03519646
What this trial is testing

Eliglustat on Gaucher Disease Type IIIB

Who this might be right for
Gaucher Disease, Type III
National Taiwan University Hospital 4
Large-scale testing (Phase 3)Active Not RecruitingNCT05222906
What this trial is testing

Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3

Who this might be right for
Gaucher's Disease Type III
Sanofi 43
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)Ended earlyNCT00199342
What this trial is testing

A Monoclonal Antibody, KW-2871, in Patients With Advanced Melanoma

Who this might be right for
Stage IV Melanoma
Kyowa Kirin, Inc. 5
Early research (Phase 1)Study completedNCT03159117
What this trial is testing

Pfizer PF-06688992 in Patients With Stage III or Stage IV Melanoma

Who this might be right for
Melanoma
Memorial Sloan Kettering Cancer Center 7
Post-approval studies (Phase 4)Study completedNCT04002830
What this trial is testing

A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

Who this might be right for
Gaucher Disease, Type 3
Ari Zimran 14
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