Testing effectiveness (Phase 2)Study completedNCT03921554What this trial is testingJAK Inhibitor Treatment in AGSWho this might be right forAicardi Goutieres Syndrome Adeline Vanderver, MD 54
Large-scale testing (Phase 3)Ended earlyNCT04517253What this trial is testingBaricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGSWho this might be right forNakajo-Nishimura SyndromeChronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature SyndromeSTING-Associated Vasculopathy With Onset in Infancy+1 more Eli Lilly and Company 10
Not applicableStudy completedNCT02699190What this trial is testingLeukoSEQ: Whole Genome Sequencing as a First-Line Diagnostic Tool for LeukodystrophiesWho this might be right forLeukodystrophyWhite Matter Disease4H Syndrome+61 more Children's Hospital of Philadelphia 236
Not applicableLooking for participantsNCT03047369What this trial is testingThe Myelin Disorders Biorepository ProjectWho this might be right forLeukodystrophyWhite Matter DiseaseLeukoencephalopathies+63 more Children's Hospital of Philadelphia 12,000