| Status | Study |
| Recruiting |
Study Name: Biomarker for Patient With Cystinosis Disease or Highly Suspected for Cystinosis Disease Condition: Cystine Storage Disease Cystinosis, Nephropathic Lysosomal Date: 2016-07-12 |
| Recruiting |
Study Name: Biomarker for Patients With Fabry Disease Condition: Fabry Disease Anderson-Fabry Disease Date: 2016-05-18 |
| Recruiting |
Study Name: LYSO-PROVE - Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease Condition: Lysosomal Storage Diseases Gaucher Disease Date: 2015-03-13 |
| Not yet recruiting |
Study Name: Diet and Exercise in Pompe Disease Condition: Pompe Disease Glycogen Storage Disease Type II Date: 2015-02-09 Interventions: Other: Diet and Exercise Exercise will consist of 2x/week |
| Recruiting |
Study Name: Investigating Lysosomal Storage Diseases in Minority Groups Condition: Lysosomal Storage Disorders Gaucher Disease Date: 2014-04-17 |
| Recruiting |
Study Name: Molecular and Cellular Mechanisms of Lysosomal Storage Diseases Condition: Lysosomal Storage Disorders Date: 2013-11-25 |
| Recruiting |
Study Name: Lysosomal Storage Disease: Health, Development, and Functional Outcome Surveillance in Preschool Children Condition: Mucopolysaccharidosis Type I (MPS I) Mucopolysaccharidosis Type II (MPS II) Date: 2013-08-06 |
| Completed |
Study Name: Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders Condition: Lysosomal Storage Disease Peroxisomal Disorder Date: 2012-06-20 Interventions: Drug: Campath-1H A daily dose |
| Active, not recruiting |
Study Name: Gene Therapy for Metachromatic Leukodystrophy Condition: Lysosomal Storage Disease Date: 2012-03-16 Interventions: Genetic: Autologous CD34+ stem cells transduced with ARSA encoding lentiviral vector Autologous hematopo |
| Recruiting |
Study Name: Biomarker for Metachromatic Leukodystrophy Disease Condition: Leukodystrophy, Metachromatic Hereditary Central Nervous System Demyelinating Diseases Date: 2012-02-21 |