246. メチルマロン酸血症
［臨床試験数：14，薬物数：22（DrugBank：2），標的遺伝子数：1，標的パスウェイ数：6］
Searched query = "Methylmalonic acidemia"
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 | NCT04581785 (ClinicalTrials.gov) | January 15, 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 | Not yet recruiting | 6 Months | 12 Years | All | 8 | Phase 1;Phase 2 | United States |