{"disease":{"id":"hereditary-inclusion-body-myopathy","name":"hereditary inclusion body myopathy"},"drugs":{"marketed":[],"pipeline":[],"offLabel":[],"totalMarketed":0,"totalPipeline":0},"trials":{"data":[{"nct_id":"NCT01784679","title":"GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)","phase":"","overall_status":"COMPLETED","enrollment_count":319,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":false},{"nct_id":"NCT02736188","title":"Study to Evaluate the Safety and Efficacy of Aceneuramic Acid Extended-Release (Ace-ER) Tablets in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)","phase":"PHASE3","overall_status":"TERMINATED","enrollment_count":143,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":true},{"nct_id":"NCT02377921","title":"Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Sialic Acid in Patients With Glucosamine (UDP-N-acetyl)-2-epimerase Myopathy (GNEM) or Hereditary Inclusion Body Myopathy (HIBM)","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":89,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":true},{"nct_id":"NCT01830972","title":"An Open Label Phase 2 Extension Study of Higher Dose Sialic Acid-Extended Release (SA-ER) Tablets and Sialic Acid-Immediate Release (SA-IR) Capsules in Patients With Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy","phase":"PHASE2","overall_status":"COMPLETED","enrollment_count":59,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":true},{"nct_id":"NCT02731690","title":"A Study to Evaluate the Safety of Aceneuramic Acid Extended Release (Ace-ER; UX001) Tablets in Glucosamine (UDP-N-acetyl)-2-Epimerase (GNE) Myopathy (GNEM) (Also Known as Hereditary Inclusion Body Myopathy [HIBM]) Patients With Severe Ambulatory Impairment","phase":"PHASE2","overall_status":"TERMINATED","enrollment_count":42,"lead_sponsor_name":"Ultragenyx Pharmaceutical Inc","has_results":true},{"nct_id":"NCT01634750","title":"Phase I Clinical Trial of ManNAc in Patients With GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":26,"lead_sponsor_name":"National Human Genome Research Institute (NHGRI)","has_results":false},{"nct_id":"NCT04671472","title":"Efficacy Confirmation Study of NPC-09","phase":"PHASE3","overall_status":"COMPLETED","enrollment_count":14,"lead_sponsor_name":"Nobelpharma","has_results":false},{"nct_id":"NCT00195637","title":"Intravenous Immune Globulin to Treat Hereditary Inclusion Body Myopathy","phase":"PHASE1","overall_status":"COMPLETED","enrollment_count":4,"lead_sponsor_name":"National Human Genome Research Institute (NHGRI)","has_results":false}],"total":8},"guidelines":[],"source":"Drug Landscape verified database"}