19. Lysosomal storage disease
784 clinical trials,   673 drugs   (DrugBank: 101 drugs),   68 drug target genes,   184 drug target pathways
Searched query = "Lysosomal storage disease", "Lysosomal disease", "Gaucher disease", "Niemann-Pick disease", "Niemann-Pick type C", "GM1-gangliosidosis", "GM1-gangliosidoses", "GM2-gangliosidosis", "GM2-gangliosidoses", "Tay-Sachs disease", "Sandhoff disease", "Krabbe disease", "Metachromatic leukodystrophy", "Multiple-sulfatase deficiency", "Farber disease", "Mucopolysaccharidosis type I", "Mucopolysaccharidosis I", "MPS I", "Hurler syndrome", "Scheie syndrome", "Mucopolysaccharidosis type II", "Mucopolysaccharidosis II", "MPS II", "Hunter syndrome", "Mucopolysaccharidosis type III", "Mucopolysaccharidosis III", "MPS III", "Sanfilippo syndrome", "Mucopolysaccharidosis type IV", "Mucopolysaccharidosis IV", "MPS IV", "MPS IVA", "Morquio syndrome", "Morquio A syndrome", "Mucopolysaccharidosis type VI", "Mucopolysaccharidosis VI", "MPS VI", "Maroteaux-Lamy syndrome", "Mucopolysaccharidosis type VII", "Mucopolysaccharidosis VII", "MPS VII", "Sly syndrome", "Mucopolysaccharidosis type IX", "Mucopolysaccharidosis IX", "MPS IX", "Hyaluronidase deficiency", "Sialidosis", "Galactosialidosis", "Mucolipidosis II", "Mucolipidosis type II", "I-cell disease", "Mucolipidosis III", "Mucolipidosis type III", "Alpha-Mannosidosis", "Alpha-Mannosidase Deficiency", "Beta-Mannosidosis", "Beta-Mannosidase Deficiency", "Fucosidosis", "Aspartylglucosaminuria", "Schindler disease", "Kanzaki disease", "Pompe disease", "Acid lipase deficiency", "Wolman disease", "Cholesterol ester storage disease", "Danon disease", "Free sialic acid storage disease", "Salla disease", "Ceroid lipofuscinosis", "Fabry disease", "Cystinosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
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 |
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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 |