A Screening Study Evaluating Disease Status of Gaucher Type I Patients

Biomarkers Related to Bone in Pediatric Gaucher Disease

NCT06116071 ·Status: RECRUITING

International Collaborative Gaucher Group (ICGG) Gaucher Disease Registry & Pregnancy Sub-registry

NCT00358943 ·Status: RECRUITING

A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients

NCT00358150 ·Status: COMPLETED ·Phase: PHASE2

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

Assessment Of Liver and Spleen Fibrosis in Patients With Gaucher Disease Using Fibroscan

NCT01898325 ·Status: UNKNOWN ·Phase: NA

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

NCT00553631 ·Status: COMPLETED ·Phase: PHASE3

Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease

NCT02416661 ·Status: COMPLETED

Phase II Study of Glucocerebrosidase in Patients With Gaucher Disease

NCT00004293 ·Status: UNKNOWN ·Phase: PHASE2

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

NCT00430625 ·Status: COMPLETED ·Phase: PHASE3

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

NCT00635427 ·Status: COMPLETED ·Phase: PHASE3

Natural History Study of Infantile and Juvenile GM1 Gangliosidosis (GM1) Patients

NCT04041102 ·Status: COMPLETED

A Gene Therapy Study in Patients With Gaucher Disease Type 1

NCT05324943 ·Status: COMPLETED ·Phase: PHASE1

Implementation and Evaluation of a Rare Disease Algorithm to Identify Persons at Risk of Gaucher Disease Using Data From Electronic Health Records (EHRs) in the United States (Project Searchlight)

NCT05908656 ·Status: COMPLETED ·Phase: NA

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

NCT04836377 ·Status: TERMINATED

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

NCT04470713 ·Status: COMPLETED

Prospective Collection of Biospecimen in Pediatric Patients and Adult Guardians Diagnosed With Glycogen Storage Disease Type 1B (GSD1b)

NCT05915910 ·Status: TERMINATED

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

NCT00364858 ·Status: COMPLETED ·Phase: PHASE4

Study to Evaluate Blood Cell Lines From Patients With Gaucher Disease

NCT00351156 ·Status: COMPLETED

GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)

NCT05109793 ·Status: COMPLETED

A Gaucher Disease Gene Therapy Trial With FLT201

NCT07223944 ·Status: RECRUITING ·Phase: PHASE3

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

NCT02605603 ·Status: UNKNOWN

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

NCT04145037 ·Status: TERMINATED ·Phase: PHASE1/PHASE2

A Study in Type 1 Gaucher Patients to Evaluate the Pharmacokinetics, Safety and Pharmacodynamics of AT2101

NCT00875160 ·Status: TERMINATED ·Phase: PHASE1

Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease

NCT05447494 ·Status: UNKNOWN ·Phase: PHASE1/PHASE2

Macrophagic Activation and Immunophenotypic Profile of Patients With Gaucher's Disease

NCT01206673 ·Status: COMPLETED ·Phase: NA