113. Muscular dystrophy Clinical trials / Disease details
Clinical trials : 646 / Drugs : 471 - (DrugBank : 105) / Drug target genes : 59 - Drug target pathways : 170
| 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-jRCT2031210252 | 06/09/2021 | 18/08/2021 | A phase 1 study in patients with Fukuyama-type congenital muscular dystrophy | A multicenter phase 1 study of NS-035 in patients with Fukuyama-type congenital muscular dystrophy | Fukuyama-type congenital muscular dystrophy | This study consists of the following 4 cohorts. The study will start with cohort 1 and only D-mannitol will be administered once during the premedication phase, followed by 12 simultaneous doses of NS-035 and D-mannitol once weekly during the treatment phase. The dose of D-mannitol was fixed at 500 mg / kg in all cohorts, and NS-035 was gradually increased from cohort 1 to cohort 4 (1.6 mg/kg, 6.0 mg/kg, 20 mg/kg and 40 mg/kg, respectively). | Toda Tatsushi | NULL | Recruiting | >= 5age old | <= 10age old | Both | 12 | Phase 1 | Japan |