113. 筋ジストロフィー Muscular dystrophy Clinical trials / Disease details
臨床試験数 : 646 / 薬物数 : 471 - (DrugBank : 105) / 標的遺伝子数 : 59 - 標的パスウェイ数 : 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 | NCT05230459 (ClinicalTrials.gov) | March 12, 2023 | 27/1/2022 | A Study to Evaluate the Safety of LION-101 in Subjects With Genetic Confirmation of LGMD2I/R9 (Part1) | A Phase 1/2, Double-blind, Randomized, Placebo-controlled, Dose-escalation Study to Evaluate the Safety of LION-101 Gene Therapy in Adult Subjects (18-65 Years) With Genetic Confirmation of Limb Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) - Part 1 | Limb Girdle Muscular Dystrophy;Limb-Girdle Muscular Dystrophy Type 2;LGMD2I;Muscular Dystrophy;LGMD2;LGMD;FKRP;FKRP Mutation;Fukutin Related Protein | Genetic: LION-101 dose level 1;Genetic: LION-101 dose level 2;Other: Placebo | Asklepios Biopharmaceutical, Inc. | NULL | Recruiting | 18 Years | 65 Years | All | 10 | Phase 1/Phase 2 | United States |