30. Distal myopathy
13 clinical trials, 16 drugs (DrugBank: 3 drugs), 1 drug target gene, 1 drug target pathway
Searched query = "Distal myopathy", "Distal muscular dystrophy", "Miyoshi myopathy", "Distal dysferlinopathy", "DMRV/GNE myopathy", "Oculopharyngodistal myopathy"
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 |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | JPRN-jRCT2021200030 | 11/01/2021 | 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 | Pending | >= 20age old | <= 50age old | Both | 10 | Phase 3 | Japan |
| 2 | 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;Phase 3 | Japan |
| 3 | 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) | 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;Phase 3 | Japan | |
| 4 | 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 |