19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT02583672 (ClinicalTrials.gov) | September 2015 | 21/8/2015 | Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1) | Role of Oxidative Stress and Inflammation in Type 1 Gaucher Disease (GD1): Potential Use of Antioxidant/Anti-inflammatory Medications | Gaucher Disease Type 1 | Drug: N-acetylcysteine | University of Minnesota | Rare Diseases Clinical Research Network;National Center for Advancing Translational Sciences (NCATS);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);National Institute of Neurological Disorders and Stroke (NINDS);Lysosomal Disease Network | Recruiting | 18 Years | N/A | All | 50 | Phase 2 | United States |
| 2 | NCT01427517 (ClinicalTrials.gov) | July 2011 | 30/8/2011 | Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease | Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease | Parkinson's Disease;Gaucher's Disease | Drug: N-acetylcysteine | University of Minnesota | Rare Diseases Clinical Research Network;National Center for Advancing Translational Science (NCATS);National Institute of Neurological Disorders and Stroke (NINDS);National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) | Completed | 18 Years | N/A | All | 9 | Phase 1 | United States |
| 3 | NCT00028262 (ClinicalTrials.gov) | February 2001 | 17/12/2001 | Cystagon to Treat Infantile Neuronal Ceroid Lipofuscinosis | A Combination Therapy With Cystagon and N-Acetylcysteine for INCL Patients | Infantile Neronal Ceroid Lipofuscinosis | Drug: Cystagon | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) | NULL | Completed | 6 Months | 3 Years | All | 10 | Phase 4 | United States |