Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism

CompletedOBSERVATIONAL
Enrollment

69

Participants

Timeline

Start Date

August 1, 2019

Primary Completion Date

August 29, 2020

Study Completion Date

October 16, 2020

Conditions
Inborn Errors of MetabolismUrea Cycle DisorderMaple Syrup Urine DiseasePhenylketonuriasMethylmalonicacidemiaOTC DeficiencyAminoacidopathyPatient Reported Outcome Measurements
Interventions
OTHER

Survey

Survey: 38 preselected patient and parent reported outcomes; rating scale: 9 point likert scale (1=not at all important - 9=very important)

Trial Locations (1)

8032

University Children's Hospital Zurich, Zurich

All Listed Sponsors
lead

University Children's Hospital, Zurich

OTHER

NCT04248062 - Patient and Observer Reported Outcome Measurements in Inborn Errors of Metabolism | Biotech Hunter | Biotech Hunter