In short
Clinical trials of Sapropterin Dihydrochloride are studying people with phenylketonuria, also called PKU. These trials look at how well it works compared with another study drug and measure changes in blood phenylalanine (Phe) levels. The main goal is to understand efficacy in participants age 2 years and older.
Key points
- Clinical trials of Sapropterin Dihydrochloride are focused on phenylketonuria, also called PKU. The main study is a Phase 3, randomized, crossover, open-label, active-controlled trial. It includes participants 2 years of age and older. The trial compares Sapropterin Dihydrochloride-related treatment with sepiapterin and looks at changes in blood phenylalanine, or Phe, levels. The study is completed and enrolled 173 participants.
Trial overview
The available trial studied phenylketonuria, also called PKU, in participants 2 years of age and older. It was a Phase 3 study and aimed to compare the effect of sepiapterin versus Sapropterin Dihydrochloride in lowering blood Phe levels.
Who participated
The study included people with PKU who were at least 2 years old. The trial data do not list more detailed entry rules, so the main known target group is children and adults with this condition.
Study design and phase
This was a randomized, crossover, open-label, active-controlled trial. 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.
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.
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. 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.
Trial status and size
The trial was completed and enrolled 173 participants. 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.