- authorisedHuman Pharmacology (Phase I)- First administration to humans
A phase 1 first-in-human study of a phenylalanine-binding polymer (phelimin) in healthy volunteers and adult, classical PKU patients
This early-stage clinical trial tests a new medicine called phelimin for phenylketonuria (PKU). It is the first time phelimin is being given to humans. Researchers want to check if it is safe and see how it works in healthy adult men and men living with classical PKU.
Denmark - authorisedTherapeutic exploratory (Phase II)
A Phase 2, Randomized, Double-blind, Placebo-controlled, Dose-ranging Study to Evaluate the Safety, Tolerability, and Efficacy of MZE782 in Adults with Phenylketonuria (PKU)
This study tests a new medicine called MZE782 for adults with phenylketonuria (PKU). Researchers want to see if different doses of this drug can safely lower levels of phenylalanine, an amino acid that builds up in the blood of people with PKU, compared to a dummy tablet.
Germany · France · Netherlands - 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.
Denmark · Spain · Italy - recruitingTherapeutic confirmatory (Phase III)
A Phase 3, Double-blind, Placebo-controlled, Randomized Withdrawal Study to Evaluate the Efficacy and Safety of SYNB1934 in Patients with PKU (SYNPHENY-3)
This study is testing a new medicine, SYNB1934, for people with Phenylketonuria (PKU). We want to see how well it lowers a substance called phenylalanine (Phe) in the blood and if it's safe to use. It's a key step to potentially making this new treatment available.
Denmark - authorisedHuman Pharmacology (Phase I)- Other
Evaluation of AG-181 in Subjects 18 to 69 Years of Age With Phenylketonuria
This study is looking at a new medicine called AG-181 for people aged 18 to 69 with phenylketonuria, also known as PKU. It's an early-stage study to understand how the medicine works in the body.
Poland - 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.
United States · Australia · Brazil - not yet recruiting
Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience.
This study looks at how parents and medical staff are affected by finding out their baby has a rare condition called Phenylketonuria (PKU) through newborn screening. The goal is to make this process better and offer more support.
France - recruitingPHASE3
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
This study is looking at a new medicine called sepiapterin for children with Phenylketonuria (PKU). It aims to see if starting this medicine early can help protect their brain development over a long time.
United States · Australia · France - recruitingPHASE3
A Study of JNT-517 in Participants With Phenylketonuria (PKU)
This study is looking into a new tablet called JNT-517 for adults with Phenylketonuria (PKU). We want to see if it's safe, how well it works, and how the body handles it. You might take JNT-517 or a dummy pill called a placebo.
United States · Australia · Canada - recruitingNA
GMP Powdered Substitutes in PKU and TYR
This study looks at new powdered protein drinks for people with Phenylketonuria (PKU) and Tyrosinemia (TYR). These drinks, made from a natural milk ingredient called GMP, aim to be better-tasting and easier to stick with, while still managing the condition effectively.
United Kingdom - recruiting
Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU
This study looks at how a special diet for children with PKU affects their gut health. It compares children on the diet alone with those also taking medicines, and healthy siblings, to understand how different treatments change the 'good' bacteria in their tummies.
United Kingdom