246. メチルマロン酸血症 Methylmalonic acidemia Clinical trials / Disease details
臨床試験数 : 19 / 薬物数 : 26 - (DrugBank : 3) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23
| 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 | NCT04581785 (ClinicalTrials.gov) | May 29, 2021 | 2/10/2020 | Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia | A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations | Methylmalonic Acidemia | Biological: hLB-001 | LogicBio Therapeutics, Inc. | NULL | Recruiting | 6 Months | 12 Years | All | 8 | Phase 1/Phase 2 | United States;Saudi Arabia |