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Results for “Gaucher”

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Showing 20 of 0 results

Large-scale testing (Phase 3)Study completedNCT00319046
What this trial is testing

Clinical Study to Evaluate the Long Term Efficacy, Safety and Tolerability of Miglustat in Patients With Stable Type 1 Gaucher Disease

Who this might be right for
Gaucher Disease Type 1
Actelion 42
Not applicableLooking for participantsNCT05992532
What this trial is testing

GammaGA: Prevalence of Acid Sphingomyelinase Deficiency Disease (ASMD) and Gaucher Disease in Patients With Monoclonal Gammopathies and/or Multiple Myeloma

Who this might be right for
Gaucher DiseaseAcid SphingoMyelinase Deficiency
Fundación Española de Hematología y Hemoterapía 210
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
Not applicableUnknownNCT03590548
What this trial is testing

Clinical Audit of Managment of Gausher Disease in Children

Who this might be right for
Gaucher Disease
Yasmeen Abd Elbaset Hamzaa 1
Not applicableEnded earlyNCT04094181
What this trial is testing

VPRIV in Participants With Gaucher Disease Previously Treated With Other Enzyme Replacement Therapies or Substrate Reduction Therapies

Who this might be right for
Gaucher Disease
Shire 2
Not applicableEnrolling By InvitationNCT00001215
What this trial is testing

Genetic Studies of Lysosomal Storage Disorders

Who this might be right for
Lysosomal Storage DisordersGaucher DiseaseParkinson Disease
National Human Genome Research Institute (NHGRI) 1,000
Not applicableAvailableNCT07675031
What this trial is testing

Managed Access Program of Venglustat in Pediatric and Adult Patients With Type 3 Gaucher Disease (GD3).

Who this might be right for
Type 3 Gaucher Disease
Sanofi
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 participantsNCT07758816
What this trial is testing

Advancing Integrated Therapies for Gaucher Disease

Who this might be right for
Gaucher Disease
Yale University 30
Not applicableStudy completedNCT01272687
What this trial is testing

Study to Determine Mutations in the Gaucher Gene in Patients With Idiopathic Parkinson's Disease for Phenotype-genotype Correlation

Who this might be right for
Parkinson DiseaseIdiopathic Parkinson Disease
CENTOGENE GmbH Rostock 1,500
Large-scale testing (Phase 3)Study completedNCT01614574
What this trial is testing

Study of Velaglucerase Alfa Enzyme Replacement Therapy in Japanese Patients With Gaucher Disease

Who this might be right for
Gaucher Disease
Shire 6
Not applicableNot Yet RecruitingNCT06258577
What this trial is testing

Screening for Gaucher Disease and Acid Sphingomyelinase Deficiency

Who this might be right for
Gaucher Disease
Chung-Hsing Wang 50
Testing effectiveness (Phase 2)Study completedNCT02107846
What this trial is testing

An Open-Label, Dose Escalation Study to Evaluate the Safety and the Pharmacokinetics of Oral PRX-112

Who this might be right for
Gaucher Disease
Protalix 10
Post-approval studies (Phase 4)UnknownNCT03721627
What this trial is testing

Chronic Hepatitis C Treatment in Egyptian Children With Gaucher Disease.

Who this might be right for
Gaucher DiseaseHCV
Mansoura University Children Hospital 10
Not applicableStudy completedNCT00302146
What this trial is testing

Positron Emission Tomography (PET) Imaging in People With Gaucher Mutations

Who this might be right for
Parkinson DiseaseGaucher Disease
National Human Genome Research Institute (NHGRI) 64
Not applicableLooking for participantsNCT03190837
What this trial is testing

A Long-term Follow-up Study of Gaucher Disease

Who this might be right for
Gaucher Disease
Duke University 200
Not applicableLooking for participantsNCT00029965
What this trial is testing

Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders

Who this might be right for
Neurological RegressionMyoclonusCherry Red Spot+1 more
National Human Genome Research Institute (NHGRI) 200
Large-scale testing (Phase 3)Study completedNCT00705939
What this trial is testing

Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Who this might be right for
Gaucher Disease
Pfizer 45
Not applicableWithdrawnNCT04189601
What this trial is testing

Complement Activation in the Lysosomal Storage Disorders

Who this might be right for
Fabry DiseaseGaucher DiseaseNiemann-Pick Disease, Type C+1 more
Melbourne Health
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
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