- Active not recruitingPHASE3
A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria
This study looks at the long-term safety of a new medicine, PTC923, for people with Phenylketonuria (PKU). It also checks if the medicine helps people eat more varied foods by seeing if their usual diet and protein intake change over time.
For: PhenylketonuriaUnited States · Australia · Brazil - Ongoing, recruitingTherapeutic confirmatory (Phase III)
A Phase 3, Randomized, Crossover, Open-Label, Active-Controlled Study of Sepiapterin versus Sapropterin in Participants With Phenylketonuria ≥2 years of Age
This study looks at a new medicine called sepiapterin for people aged 18 and over with phenylketonuria (PKU). It compares sepiapterin to an existing medicine, sapropterin, to see how well it lowers levels of a substance called phenylalanine in the blood, which is important for managing PKU.
For: PhenylketonuriaDenmark · Spain · Italy