- not yet recruitingNA
PKU Microtablets Case Studies
This study looks at new PKU Microtablets, which are a different way for people with Phenylketonuria (PKU) to get their protein. We want to see how well people tolerate them, how easy they are to use, and if they are safe for children (from age 4) and adults.
United Kingdom - recruiting
A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
This study looks at the long-term safety of a medicine called pegvaliase for people with Phenylketonuria (PKU). It helps us understand side effects and how well safety precautions work over 10 years.
United States · Germany · Italy - active not recruitingPHASE1, PHASE2
AAV Gene Therapy Study for Subjects with PKU
This study is testing a new gene therapy called BMN 307 for Phenylketonuria (PKU). Researchers want to see if it's safe and helps adults with PKU by giving them a single treatment. It’s an early-stage study to understand how well it works.
United States · United Kingdom - enrolling by invitation
A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability
This study is testing a new home blood testing device for people with Phenylketonuria (PKU). It's called the Egoo Phe System. We want to see how accurate it is compared to current tests and how easy it is for people to use at home.
United Kingdom - active not recruitingPHASE3
Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria
This study looks at a new injection called pegvaliase for teenagers aged 12-17 with PKU whose current treatment isn't quite managing their condition. It aims to see if pegvaliase is safe and works better than just diet for controlling PKU.
United States · Germany