Phenylketonuria
Conditions
Brief summary
Mean change in full-scale intelligence quotient (FSIQ) (WPPSI-IV or WISC-V) over a 2-year period: WPPSI-IV (Wechsler Preschool and Primary Scale of Intelligence - Fourth Edition): for participants ≥30 months to <6 years of age, WISC-V (Wechsler Intelligence Scale for Children - Fifth Edition): for participants ≥6 years to 16 years of age
Detailed description
01. Change from baseline in phenylketonuriaquality of life (PKU-QOL) questionnaire score over time, 02. Change from baseline in European Quality of Life - 5 Dimensions (EQ-5D) score over time, 03. Mean change in FSIQ (WPPSI-IV or WISC-V) over a 4-year period, 04. Change of phenylalanine (Phe) levels over time
Interventions
DRUGPTC923
Sponsors
PTC Therapeutics Inc.
Eligibility
Sex/Gender
All
Age
0 Years to 17 Years
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| 01. Change from baseline in phenylketonuriaquality of life (PKU-QOL) questionnaire score over time, 02. Change from baseline in European Quality of Life - 5 Dimensions (EQ-5D) score over time, 03. Mean change in FSIQ (WPPSI-IV or WISC-V) over a 4-year period, 04. Change of phenylalanine (Phe) levels over time | — |
Primary
| Measure | Time frame |
|---|---|
| Mean change in full-scale intelligence quotient (FSIQ) (WPPSI-IV or WISC-V) over a 2-year period: WPPSI-IV (Wechsler Preschool and Primary Scale of Intelligence - Fourth Edition): for participants ≥30 months to <6 years of age, WISC-V (Wechsler Intelligence Scale for Children - Fifth Edition): for participants ≥6 years to 16 years of age | — |
Countries
France, Ireland, Poland, Sweden
Outcome results
None listed