Survey Study for Velaglucerase Alfa (VPRIV) in Japan

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

NCT04718779 ·Status: COMPLETED ·Phase: PHASE4

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)

NCT03333447 ·Status: COMPLETED

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

NCT04120506 ·Status: COMPLETED ·Phase: PHASE4

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

NCT03702361 ·Status: COMPLETED ·Phase: PHASE4

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

NCT01685216 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

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

NCT00635427 ·Status: COMPLETED ·Phase: PHASE3

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

NCT00430625 ·Status: COMPLETED ·Phase: PHASE3

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

NCT01842841 ·Status: COMPLETED ·Phase: PHASE3

The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease

NCT02528617 ·Status: WITHDRAWN ·Phase: PHASE4

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

NCT02843035 ·Status: ACTIVE_NOT_RECRUITING ·Phase: PHASE2

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

NCT00391625 ·Status: COMPLETED ·Phase: PHASE1/PHASE2

A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2

NCT04221451 ·Status: TERMINATED ·Phase: PHASE3

A Screening Study Evaluating Disease Status of Gaucher Type I Patients

NCT00795197 ·Status: WITHDRAWN

Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

NCT00962260 ·Status: NO_LONGER_AVAILABLE

Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

NCT05710692 ·Status: RECRUITING ·Phase: PHASE2/PHASE3

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

NCT04656600 ·Status: COMPLETED ·Phase: PHASE4

A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)

NCT05543174 ·Status: COMPLETED ·Phase: PHASE3

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

NCT01422187 ·Status: COMPLETED ·Phase: PHASE3

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

NCT00954460 ·Status: APPROVED_FOR_MARKETING

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

NCT01136304 ·Status: COMPLETED

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

NCT01411228 ·Status: COMPLETED ·Phase: PHASE3

Identification of Undiagnosed Gaucher Disease

NCT01716741 ·Status: UNKNOWN ·Phase: NA

Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease

NCT00364858 ·Status: COMPLETED ·Phase: PHASE4

Phase I Single Dose-Escalation Safety Study of Human Glucocerebrosidase (prGCD)

NCT00258778 ·Status: COMPLETED ·Phase: PHASE1

A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease

NCT06150820 ·Status: ACTIVE_NOT_RECRUITING ·Phase: NA