30. 遠位型ミオパチー Distal myopathy Clinical trials / Disease details
臨床試験数 : 15 / 薬物数 : 17 - (DrugBank : 3) / 標的遺伝子数 : 1 - 標的パスウェイ数 : 1
| 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 | JPRN-jRCT2021200030 | 08/02/2021 | 07/12/2020 | Efficacy confirmation study of NPC-09 | Efficacy confirmation study of NPC-09 - NPC-09-1 | Distal myopathy with rimmed vacuoles (DMRV), hereditary inclusion body myopathy (hIBM) | Oral administration of NPC-09 (Aceneuramic acid) for 48 weeks as compared with placebo | Suzuki Ryoichi | NULL | Not Recruiting | >= 20age old | <= 50age old | Both | 10 | Phase 3 | Japan |
| 2 | NCT04671472 (ClinicalTrials.gov) | February 8, 2021 | 30/11/2020 | Efficacy Confirmation Study of NPC-09 | Efficacy Confirmation Study of NPC-09 | GNE Myopathy;Distal Myopathy With Rimmed Vacuoles (DMRV);Hereditary Inclusion Body Myopathy (hIBM);Nonaka Disease | Drug: NPC-09;Drug: NPC-09 placebo | Nobelpharma | NULL | Completed | 20 Years | 50 Years | All | 14 | Phase 3 | Japan |
| 3 | JPRN-UMIN000026354 | 2017/03/28 | 20/03/2017 | A Phase II/III Open-label Extension Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) | A Phase II/III Open-label Extension Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) - A Phase II/III Open-label Extension Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) | GNE myopathy (Distal myopathy with rimmed vacuoles (DMRV), hereditary inclusion body myopathy (hIBM) or Nonaka disease) | SA-ER 2g 3 times oral dosing a day for 48 consecutive weeks | Tohoku University Hospital, Department of Neurology | Nobelpharma Co., Ltd. | Complete: follow-up complete | 20years-old | 50years-old | Male and Female | 20 | Phase 2,3 | Japan |
| 4 | JPRN-UMIN000020683 | 2016/02/01 | 25/01/2016 | A Phase II/III Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) | A Phase II/III Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) - A Phase II/III Study of NPC-09 (N-acetylneuraminic acid) in Patients with GNE Myopathy (GNEM) | GNE myopathy (Distal myopathy with rimmed vacuoles (DMRV), hereditary inclusion body myopathy (hIBM) or Nonaka disease) | SA-ER 2g 3 times oral dosing a day for 48 consecutive weeks placebo 3 times oral dosing a day for 48 consecutive weeks | Tohoku University Hospital, Department of Neurology | Nobelpharma Co., Ltd. | Complete: follow-up complete | 20years-old | 50years-old | Male and Female | 20 | Phase 2,3 | Japan |
| 5 | NCT01236898 (ClinicalTrials.gov) | November 2010 | 8/11/2010 | Pharmacokinetic Study on N-acetylneuraminic Acid | Pharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM) | Nonaka Myopathy;Hereditary Inclusion Body Myopathy | Drug: NPC-09 | Tohoku University | NULL | Completed | 20 Years | 40 Years | Both | 6 | Phase 1 | Japan |