A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin (EPIPHENY)
Phase 3 – large-scale trial before approval
Conditions:
Phenylketonuria
Sponsor: PTC Therapeutics Inc.
trial.available_in:
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Overview
Trial status: Authorised
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
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
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
Who can participate
Age group: 0-17 years
Gender: Female, Male
Target enrollment: 60 participants
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