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 | NCT04836494 (ClinicalTrials.gov) | March 25, 2021 | 29/3/2021 | A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia | A First-in-human, Randomized, Placebo-controlled, Single and Multiple Ascending Dose Escalation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BBP-671 in Healthy Subjects and In Patients With Propionic Acidemia or Methylmalonic Acidemia | Healthy Volunteers;Propionic Acidemia;Methylmalonic Acidemia;Organic Acidemia | Drug: BBP-671;Drug: Placebo | CoA Therapeutics, Inc., a BridgeBio company | NULL | Recruiting | 18 Years | 55 Years | All | 128 | Phase 1 | United States |