156. レット症候群 Rett syndrome Clinical trials / Disease details
臨床試験数 : 40 / 薬物数 : 53 - (DrugBank : 19) / 標的遺伝子数 : 77 - 標的パスウェイ数 : 113
| 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 | NCT03059160 (ClinicalTrials.gov) | April 1, 2017 | 2/2/2017 | Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. | Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome. | Rett Syndrome | Drug: Tridecanoic Acid | Sheba Medical Center | Ultragenyx Pharmaceutical Inc | Not yet recruiting | 5 Years | 18 Years | Female | 10 | Phase 2 | NULL |
| 2 | NCT02696044 (ClinicalTrials.gov) | June 2016 | 25/2/2016 | Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin | Treatment of Mitochondrial Dysfunction in Rett Syndrome With Triheptanoin: An Open-label, 10-subject Clinical Trial of UX007 (Triheptanoin) in the Treatment of Mitochondrial Dysfunction in Participants With Rett Syndrome, Dyskinesia, and Epilepsy | Rett Syndrome | Drug: triheptanoin | Center for Rare Neurological Diseases, Norcross, GA | Ultragenyx Pharmaceutical Inc;Rett Syndrome Research Trust | Recruiting | 2 Years | N/A | Female | 12 | Phase 2 | United States |