156. Rett syndrome Clinical trials / Disease details


Clinical trials : 44 Drugs : 61 - (DrugBank : 23) / Drug target genes : 57 - Drug target pathways : 83

  
2 trials found
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1NCT03059160
(ClinicalTrials.gov)
April 1, 20172/2/2017Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.Open Label Trial of Triheptanoin (UX007) in Treatment of Rett Syndrome.Rett SyndromeDrug: Tridecanoic AcidSheba Medical CenterUltragenyx Pharmaceutical IncNot yet recruiting5 Years18 YearsFemale10Phase 2NULL
2NCT02696044
(ClinicalTrials.gov)
June 201625/2/2016Treatment of Mitochondrial Dysfunction in Rett Syndrome With TriheptanoinTreatment 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 EpilepsyRett SyndromeDrug: triheptanoinCenter for Rare Neurological Diseases, Norcross, GAUltragenyx Pharmaceutical Inc;Rett Syndrome Research TrustRecruiting2 YearsN/AFemale12Phase 2United States