Table of Contents
Trial overview
The available trial studied phenylketonuria, also called PKU, in participants 2 years of age and older.[1] It was a Phase 3 study and aimed to compare the effect of sepiapterin versus Sapropterin Dihydrochloride in lowering blood Phe levels.[1]
Who participated
The study included people with PKU who were at least 2 years old.[1] The trial data do not list more detailed entry rules, so the main known target group is children and adults with this condition.[1]
Study design and phase
This was a randomized, crossover, open-label, active-controlled trial.[1] Randomized means treatment assignment was by chance, crossover means participants received more than one treatment during the study, open-label means everyone knew which treatment was given, and active-controlled means the study compared one treatment with another treatment.[1]
The trial was in Phase 3, which is a later stage of clinical research used to study treatment effects in a larger group of participants.[1]
What was measured
The main outcome was the mean change in blood Phe levels from baseline to Weeks 3 and 4 of each treatment period, using the average of the last 2 weeks of each period.[1] In simple terms, the study wanted to see how much blood Phe changed after treatment and whether one treatment lowered it more than the other.[1]
Trial status and size
The trial was completed and enrolled 173 participants.[1] The brief summary states that the purpose was to compare the efficacy of sepiapterin to Sapropterin Dihydrochloride in reducing blood Phe levels in people with PKU.[1]
