A Phase 3 Study of PTC923 in Subjects with Phenylketonuria
Study ID: STU-2021-0692
Summary
This is a Phase 3 open-label extension study for subjects with PKU who complete a Phase 3 PTC Therapeutics (PTC)-sponsored feeder study (including Study PTC923-MD-003-PKU). The study consists consists of open-label treatment with PTC923 administered orally once a day for a minimum of 12 months or until subject experiences lack of efficacy, adverse events AEs) that lead to discontinuation, withdraws from treatment, or PTC923 receives regulatory approval.
Primary: To evaluate the long-term safety of PTC923 in subjects with phenylketonuria (PKU) Secondary: To evaluate PTC923 effect on quality of life (QOL) using the Phenylketonuria-quality of life (PKU-QOL) questionnaire in the subset of subjects that are able to complete the PKU-QOL; To evaluate PTC923 effect of QOL using the European Quality of Life 5 Dimensions (EQ-5D) Exploratory: To evaluate changes in blood tyrosine (Tyr) over time