Search clinical trials for: Neuronopathic Gaucher Disease

Total 382304 results

Takeda

Active, not recruiting

A Study of Velaglucerase Alfa (VPRIV) in Chinese Children, Teenagers, and Adults With Type 1 Gaucher Disease

Gaucher Disease

China
Sanofi

Active, not recruiting

Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 (ELIKIDS)

Gaucher's Disease Type III | Gaucher's Disease Type I

Canada, Argentina, France, Italy, Japan, Russian Federation, Spain, Sweden, Turkey, United Kingdom
Shire

Terminated

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

Gaucher Disease

Canada
Sanofi

Completed

Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ (SEED)

Gaucher's Disease

China
AVROBIO

Terminated

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

Gaucher Disease

United States, Canada
Ain Shams University

Completed

Role of Vitamin E in Gaucher Disease Patients

Gaucher Disease

Egypt
Takeda

Active, not recruiting

A Study Measuring Substances Potentially Indicating Bone Problems in Adults With Type 1 Gaucher Condition

Gaucher Disease

Spain
Genzyme, a Sanofi Company

Active, not recruiting

Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension (LEAP)

Gaucher Disease Type 1 | Gaucher Disease Type 3

Germany, United States, Japan, United Kingdom
Sanofi

Completed

A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers

Gaucher's Disease

United States
Sanofi

Completed

A Study to Evaluate Pharmacokinetic Parameters and Safety of Eliglustat Absorption Through the Mouth (Acronym)

Gaucher's Disease

United States
Takeda

Completed

A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy (SWITCH)

Gaucher Disease

United States
Sanofi

Completed

A Study to Evaluate Absolute Bioavailability, Absorption, Metabolism, and Excretion of Genz-112638 in Healthy Male Participants

Gaucher's Disease Type I
Hoffmann-La Roche

Terminated

Study for Participants With Ulcerative Colitis Previously Enrolled in Etrolizumab Phase II/III Studies (COTTONWOOD)

Ulcerative Colitis

Israel, Spain, United States, United Kingdom, Belgium, Korea, Republic of, Canada, Australia, Austria, Czechia, France, Italy, Netherlands, New Zealand, Russian Federation, Hungary, Singapore, India, Portugal, Serbia, Hong Kong, B... and more
Hadassah Medical Organization

Completed

A Second-generation AI Based Therapeutic Regimen in Patients With Gaucher Disease Treated With Enzyme Replacement Therapy.

Gaucher Disease Type 1

Israel
CENTOGENE GmbH Rostock

Active, not recruiting

Omics Gaucher Study: Multiomic Approach (OmicsGaucher)

Gaucher Disease

Germany
AVROBIO

Terminated

A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

Type 1 Gaucher Disease

United States, Canada
Shire

Completed

Post Marketing Surveillance (PMS) Study for Velaglucerase Alfa (VPRIV) in India

Gaucher Disease

India
Albert Einstein College of Medicine
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program (ScreenPlus)

Mucopolysaccharidosis II | Fabry Disease | Niemann-Pick Disease, Type C | Mucopolysaccharidosis VI | Lysosomal Acid Lipase Deficiency | Metachromatic Leukodystrophy | Gaucher Disease | Cerebrotendinous Xanthomatosis | Mucopolysaccharidosis IV A | GM1 Gangliosidosis | Mucopolysaccharidosis VII | Acid Sphingomyelinase... and other conditions

United States
Pfizer

Completed

A Multicenter Extension Study of Taliglucerase Alfa in Adult Subjects With Gaucher Disease

Gaucher Disease
HaEmek Medical Center, Israel

Terminated

Liver and the Spleen Stifness in Adult Patients With Gaucher's Disease Using Ultrasound Shear Wave Elastography

Liver Fibrosis | Gaucher Disease | Spleen; Fibrosis

Israel
Shaare Zedek Medical Center

Completed

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

Gaucher Disease, Type 1

Israel
CENTOGENE GmbH Rostock

Withdrawn

Biomarker for Gaucher Disease: BioGaucher (BioGaucher) (BioGaucher)

Splenomegaly | Hepatomegaly

Germany, India, Sri Lanka
KemPharm Denmark A/S

Terminated

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

Gaucher Disease, Type 1 | Gaucher Disease, Type 3

India
Shaare Zedek Medical Center
Shire

Completed

Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)

Gaucher Disease, Type 1
Shaare Zedek Medical Center
Pfizer

Completed

Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs

Gaucher Disease, Type 1
New York Medical College

Completed

Human Placental-Derived Stem Cell Transplantation (HPDSC)

Mucopolysaccharidosis I | Myelodysplastic Syndrome | Acute Lymphocytic Leukemia | Acute Myelogenous Leukemia | Niemann-Pick Disease | Mucopolysaccharidosis VI | Wolman Disease | Adrenoleukodystrophy | Batten Disease | Metachromatic Leukodystrophy | Diamond-Blackfan Anemia | Severe Aplastic Anemia | Fucosidosis | Gaucher... and other conditions

United States
National Taiwan University Hospital
Sanofi

Completed

Eliglustat on Gaucher Disease Type IIIB

Gaucher Disease, Type III

Taiwan
Ari Zimran
Pfizer

Active, not recruiting

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

Gaucher Disease, Type 3

Israel, Turkey, India
Genzyme, a Sanofi Company

Completed

Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies (EXOSKEL)

Gaucher Disease

Canada, Russian Federation, Tunisia
Sanofi

Completed

A National Study in Patients With Unexplained Splenomegaly (SMS)

Gaucher Disease | Splenomegaly

France
Shire

Completed

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

Gaucher Disease

United States, Spain, United Kingdom, Israel
Idorsia Pharmaceuticals Ltd.

Completed

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

GM2 Gangliosidosis | GM1 Gangliosidosis | Tay-Sachs Disease | Sandhoff Disease | Gaucher Disease, Type 2 | AB Variant Gangliosidosis GM2

United States, Spain, Germany, Italy, Belgium, Brazil, France, Portugal, Switzerland, United Kingdom
Hoffmann-La Roche

Completed

A Study of the Efficacy and Safety of Etrolizumab in Participants With Ulcerative Colitis Who Have Been Previously Exposed to Tumor Necrosis Factor (TNF) Inhibitors (HICKORY)

Ulcerative Colitis

Israel, Spain, United States, United Kingdom, Belgium, Korea, Republic of, Australia, Austria, France, Hungary, Italy, Netherlands, Canada, Germany, Poland, Brazil, Argentina, Czechia, Lithuania, Switzerland, Mexico, Denmark, Greece, ...
Shire

Completed

A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease

Gaucher Disease, Type 1

Israel, Paraguay, Argentina, Russian Federation, Tunisia
Shire

Completed

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

Gaucher Disease

Japan
CEN Biotech
Takeda; BioClinica, Inc.

Completed

Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa) (EIROS)

MRI | Gaucher Disease, Type 1

France
Shire

Completed

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

Gaucher Disease

Israel, Romania, Serbia
Shire
Quintiles, Inc.

Completed

Multicenter Extension Study of Velaglucerase Alfa in Japanese Patients With Gaucher Disease

Gaucher Disease

Japan
Shire

Completed

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

Gaucher Disease, Type 3

Egypt, India, Tunisia
Shire

Completed

An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

Gaucher Disease, Type 1

United States, Israel, Argentina, India, Korea, Republic of, Paraguay, Poland, Russian Federation, Spain, Tunisia, United Kingdom
Shire

Completed

Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase

Gaucher Disease

United States, Israel, United Kingdom, Poland, Spain
Shire

Completed

Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

Gaucher Disease, Type 1

Spain, Israel, United States, India, Paraguay, United Kingdom, Russian Federation, Tunisia, Argentina
Melbourne Health
Sanofi

Withdrawn

Complement Activation in the Lysosomal Storage Disorders (CATALYST)

Lysosomal Storage Diseases | Fabry Disease | Niemann-Pick Disease, Type C | Gaucher Disease

Australia
CENTOGENE GmbH Rostock

Completed

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease (LYSO-PROOF)

Lysosomal Storage Diseases | Sphingolipidoses | Gaucher Disease

Albania, Greece, India, Israel, Morocco, Pakistan, Spain
Shire

Approved for marketing

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

Gaucher Disease, Type 1

United States
CENTOGENE GmbH Rostock

Completed

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

Parkinson Disease | Idiopathic Parkinson Disease

Thailand, Germany
ISU Abxis Co., Ltd.

Completed

A Phase I Study to Compare Abcertin and EU-sourced Cerezyme® in Healthy Volunteers

Gaucher Disease

Australia
NYU Langone Health
Genzyme, a Sanofi Company

Completed

Genzyme Osteopenia/Osteoporosis Study

Gaucher Disease

United States
Shaare Zedek Medical Center

Completed

Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) in Treatment-naive Patients With Type 1 Gaucher Disease

Primary Disease

Israel
Shire

Completed

Home Therapy With VPRIV in Gaucher's Disease

Gaucher Disease

Austria, Germany

Search clinical trials for: Neuronopathic Gaucher Disease

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