Clinical Trials Browser

Recruiting × Gaucher Disease × Clear all

NCT03721068 2026-03-27

Study of CAR T-Cells Targeting the GD2 With IL-15+iCaspase9 for Relapsed/Refractory Neuroblastoma or Relapsed/Refractory Osteosarcoma

Phase 1 Recruiting

18 enrolled

CNSSarcoma

Gaucher Disease, Neuroblastoma, Osteosarcoma

NCT05992532 2026-02-11

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

Recruiting

210 enrolled

Plasma cell

Gaucher Disease, Multiple Myeloma, Niemann-Pick Diseases, Paraproteinemias, Splenomegaly

NCT06573723 2026-01-14

Institutional Registry of Rare Diseases

Recruiting

380 enrolled

CNSDermatologicEndocrineMyeloidPlasma cell

Immunoglobulin G4-Related Disease, Adrenal Hyperplasia, Congenital, Amyloidosis, Cardiomyopathy, Hypertrophic, Cushing Syndrome, Demyelinating Diseases, Gaucher Disease, Hepatolenticular Degeneration, von Hippel-Lindau Disease, Hirschsprung Disease, Hypertension, Pulmonary, Mastocytosis, Metabolism, Inborn Errors, Multiple Endocrine Neoplasia, Paraganglioma, Pheochromocytoma, Prader-Willi Syndrome, Sarcoidosis, Telangiectasia, Hereditary Hemorrhagic, Inflammatory Bowel Diseases, Neurocutaneous Syndromes, Rare Diseases, Vascular Malformations, Angioedemas, Hereditary, Eosinophilic Esophagitis

NCT06539169 2024-11-14

FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases

Recruiting

1,000 enrolled

Classical hematologyOtherPlasma cell

Alzheimer Disease, Amyloidosis, Amyotrophic Lateral Sclerosis, Anemia, Sickle Cell, Cystic Fibrosis, Ehlers-Danlos Syndrome, Gaucher Disease, Glycogen Storage Disease Type II, Huntington Disease, Myasthenia Gravis, Gangliosidosis, GM1, alpha-Thalassemia, beta-Thalassemia, Muscular Dystrophy, Duchenne, Insomnia, Fatal Familial, Rare Diseases