Clinical Trials for Phenylketonurias

AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)

NCT ID: NCT06332807

This clinical trial, called the PHEdom study, is exploring a new gene therapy called NGGT002 for adults with classic Phenylketonuria (PKU), a genetic condition that affects how the body processes a...

Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?

NCT ID: NCT05128149

This clinical trial is looking at how well people with Phenylketonuria (PKU) can stick to their treatment plans and how this affects their overall quality of life. PKU is a condition that requires ...

Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria

NCT ID: NCT06337864

This clinical trial is exploring a new treatment option for adults with Classical Phenylketonuria (PKU), a condition that affects how the body processes certain proteins. The study is looking at th...

Early Check: Expanded Screening in Newborns

NCT ID: NCT03655223

Walking Program in Fatty Liver Children With Phenylketonuria

NCT ID: NCT06718842

This clinical trial is looking at how a walking program can help children with two specific health issues: phenylketonuria (PKU) and non-alcoholic fatty liver disease. PKU is a genetic condition th...

Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)

NCT ID: NCT04404530

This clinical trial is studying the effects of a new medication called Palynziq on patients with Phenylketonuria (PKU), a genetic condition that makes it hard for the body to process an important a...

Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects

NCT ID: NCT06687733

This clinical trial is studying a new treatment called NGGT002 for adults with a condition known as classic Phenylketonuria (PKU). PKU is a genetic disorder that affects how the body processes a pr...

Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria

NCT ID: NCT06560736

This clinical trial is exploring new ways to help teenagers with Phenylketonuria (PKU) manage anxiety and depression. While most previous research has focused on medication to lower high blood phen...

Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU.

NCT ID: NCT05827536

This clinical trial is studying a new treatment called PKU GOLIKE, which is a special mixture of amino acids, to see how it affects daily fluctuations in blood levels of phenylalanine (Phe) in pati...

Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU

NCT ID: NCT05356377

Safety and Efficacy Study of NGGT002 in PKU Adult Subjects

NCT ID: NCT06061614

This clinical trial is studying a new gene therapy called NGGT002 to see if it is safe and effective for adults with Phenylketonuria (PKU), a genetic condition that affects how the body processes c...

PheCheck Feasibility Study

NCT ID: NCT05998109

The PheCheck Feasibility Study is a clinical trial aimed at testing a new tool called PheCheck™ that quickly measures phenylalanine (Phe) levels. This study compares how well PheCheck works against...

Comparison of Atherogenic Risk Factors and Efficacy of Nutritional Treatment Among Adult Phenylketonuria Patients

NCT ID: NCT04969809

This clinical trial is studying how proper nutritional treatment can help adults with a rare metabolic disorder called phenylketonuria (PKU). PKU occurs when the body cannot break down a substance ...