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

  
5 trials found
No.TrialIDDate_
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PhaseCountries
1NCT04671472
(ClinicalTrials.gov)
February 8, 202130/11/2020Efficacy Confirmation Study of NPC-09Efficacy Confirmation Study of NPC-09GNE Myopathy;Distal Myopathy With Rimmed Vacuoles (DMRV);Hereditary Inclusion Body Myopathy (hIBM);Nonaka DiseaseDrug: NPC-09;Drug: NPC-09 placeboNobelpharmaNULLCompleted20 Years50 YearsAll14Phase 3Japan
2JPRN-jRCT2021200030
08/02/202107/12/2020Efficacy confirmation study of NPC-09Efficacy 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 placeboSuzuki RyoichiNULLNot Recruiting>= 20age old<= 50age oldBoth10Phase 3Japan
3JPRN-UMIN000026354
2017/03/2820/03/2017A 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 weeksTohoku University Hospital, Department of NeurologyNobelpharma Co., Ltd.Complete: follow-up complete20years-old50years-oldMale and Female20Phase 2,3Japan
4JPRN-UMIN000020683
2016/02/0125/01/2016A 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 NeurologyNobelpharma Co., Ltd.Complete: follow-up complete20years-old50years-oldMale and Female20Phase 2,3Japan
5NCT01236898
(ClinicalTrials.gov)
November 20108/11/2010Pharmacokinetic Study on N-acetylneuraminic AcidPharmacokinetic Study on N-acetylneuraminic Acid in Patients With Distal Myopathy With Rimmed Vacuoles (DMRV) - Hereditary Inclusion Body Myopathy (hIBM)Nonaka Myopathy;Hereditary Inclusion Body MyopathyDrug: NPC-09Tohoku UniversityNULLCompleted20 Years40 YearsBoth6Phase 1Japan