Status | Study |
RECRUITING |
Study Name: Biomarkers Related to Bone in Pediatric Gaucher Disease Related to Bone Pathology in Pediatric Patients With Gaucher Disease Condition: Gaucher Disease Date: 2023-10-30 |
NOT_YET_RECRUITING |
Study Name: 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) Condition: Date: 2023-06-08 Interventions: Subjects that consent will have a single blood draw either via a finger stick or via venous blood draw (blood will be placed on a 5 spot blood card) u |
RECRUITING |
Study Name: Extracellular Vesicles as Potential Biomarkers and Therapeutic Target in Gaucher Disease Condition: Gaucher Disease Date: 2023-03-31 Interventions: no intervention, this is an observational study |
RECRUITING |
Study Name: World Data on Ambroxol for Patients With GD and GBA Related PD Condition: Gaucher Disease Date: 2020-05-10 |
Not yet recruiting |
Study Name: Pharmacokinetics, Pharmacodynamics And Safety Study Of Elelyso(tm) In Pediatric Subjects With Type 1 Gaucher Disease Condition: Type 1 Gaucher Disease Date: 2016-09-23 Interventions: Drug: Elelyso 60 units/kg All patients receive Elelyso 60 units/kg. |
Completed |
Study Name: Auto-antibodies Prevalence and CD1 Role in Gaucher Disease Condition: Gaucher Disease Date: 2016-01-05 Interventions: Genetic: genetic analyses |
Recruiting |
Study Name: SRT in Comparison to ERT on Immune Aspects and Bone Involvement in Gaucher Disease Condition: Gaucher Disease Date: 2015-09-09 |
Recruiting |
Study Name: Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1) Condition: Gaucher Disease Type 1 Date: 2015-08-21 Interventions: Drug: N-acetylcysteine 1800mg NAC twice daily (3600mg/day) orally for approximately 90 days. |
Recruiting |
Study Name: Miglustat on Gaucher Disease Type IIIB Condition: Gaucher Disease Date: 2015-08-06 Interventions: Drug: Miglustat combine miglus |
Recruiting |
Study Name: Intra-monocyte Imiglucerase Kinetics in Gaucher Disease Condition: Gaucher Disease Date: 2013-09-02 Interventions: Drug: Imiglucérase (drug) pharmacokinetics |