19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | NCT02124070 (ClinicalTrials.gov) | March 26, 2014 | 25/4/2014 | Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis | Therapeutic Effect of Recombinant Human Growth Hormone (rhGH) on the Myopathy of Cystinosis | Cystinosis;Myopathy | Drug: rh Growth Hormone | National Human Genome Research Institute (NHGRI) | NULL | Withdrawn | 18 Years | 70 Years | All | 0 | Phase 1/Phase 2 | United States |
| 2 | NCT00748969 (ClinicalTrials.gov) | November 2008 | 8/9/2008 | Clinical Trial of Growth Hormone in MPS I, II, and VI | Phase II/III, Randomized, Clinical Trial of the Effects of Nutropin AQ® on Growth and Bone Metabolism in Children With MPS I, II, and VI and Short Stature | Mucopolysaccharidosis I;Mucopolysaccharidosis II;Mucopolysaccharidosis VI | Drug: Somatropin (DNA origin) | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | NULL | Terminated | 5 Years | 17 Years | All | 2 | Phase 2/Phase 3 | United States |
| 3 | NCT00286689 (ClinicalTrials.gov) | February 3, 2006 | 1/2/2006 | Effects of Growth Hormone in Chronically Ill Children | Hurler Syndrome;Cerebral Palsy;Juvenile Rheumatoid Arthritis;Crohn Disease;HIV Infections | Drug: Growth Hormone;Procedure: Whole body Protein turnover;Procedure: DEXA scan | University of Texas Southwestern Medical Center | NULL | Withdrawn | 3 Years | 17 Years | All | 0 | N/A | United States |