Clinical trials
Phenylketonuria (PKU) clinical trials
Below are recruiting phenylketonuria (pku) clinical trials, each written for real people, not researchers. We’re tracking 18 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- NCT07672756RecruitingPhase 1
Testing PL54 injection for adults with PKU
This study tests a new injection called PL54 to see if it is safe and tolerable for adults with phenylketonuria (PKU), a condition where the body cannot break down an amino acid called phenylalanine. It is designed for people with high blood phenylalanine levels and may offer a new treatment option.
Hefei, AnhuiAges 18–55 - NCT06940193Enrolling by invitation
Egoo Phe home test for phenylalanine levels in PKU
This study tests a new home blood test (Egoo Phe) for measuring phenylalanine levels in people with PKU, as well as in healthy adults. It aims to see if it is accurate and easy to use.
BirminghamAges Up to 3 years - NCT06302348RecruitingPhase 3
Sepiapterin study for phenylketonuria
This study tests a new medicine called sepiapterin to see if it can help control blood phenylalanine levels in people with PKU. The trial aims to find a safe and effective dose for different age groups.
Indianapolis, IndianaAges Up to 9 years - NCT07694440RecruitingPhase 2
Study of MZE782 in Adults With PKU
This study tests a new oral medication (MZE782) for adults with PKU. It looks at how safe and effective the drug is, and it requires you to keep your diet stable for the entire study.
Oakland, CaliforniaAges 18–75 - NCT04404530Recruiting
Study nutrition changes from Palynziq in adults with PKU
This study looks at how taking Palynziq affects your nutrition and health in people with phenylketonuria (PKU). You might benefit by getting careful monitoring while researchers learn how the medicine changes diet-related measurements.
Atlanta, GeorgiaAges 16 years+ - NCT06971731RecruitingPhase 3
JNT-517 pill for adults with PKU
This trial tests a new pill called JNT-517 to see if it can lower high blood phenylalanine (Phe) levels in people with PKU. You may be a good fit if you have PKU, are at least 18, and can stick to a steady diet.
Los Angeles, CaliforniaAges 18 years+ - NCT07703020Enrolling by invitation
Obesity in PKU patients
This study looks at how common obesity is in adults with PKU and what factors may contribute to it.
Lausanne, Canton of VaudAges 18 years+ - NCT05813678Recruiting
Long-term safety monitoring for Palynziq in PKU
This study tracks the long-term safety of Palynziq (pegvaliase) in people with PKU as they take the medicine over time. It may help you and other patients understand long-term side effects and whether Palynziq stays safe.
Aurora, ColoradoAges Any age - NCT06305234Recruiting
Long-term immune response study for Palynziq PKU treatment
This long-term study checks how the immune system reacts to Palynziq (a treatment for PKU) over time. It involves extra blood tests for people already in another PKU study called 165-501.
Aurora, ColoradoAges Any age - NCT06628128RecruitingPhase 3
Long-term study of JNT-517 for people with PKU
This study tests a new medicine called JNT-517 for people with phenylketonuria (PKU) over a long period. It aims to see if the drug can help manage PKU safely and effectively.
Gainesville, FloridaAges 4 years+ - NCT06969209Recruiting
Brain aging in adults with PKU
This study looks at how the brain ages in adults with PKU. It uses an MRI scan to compare brain changes between people with PKU and healthy volunteers.
BernAges 18 years+ - NCT07484945Recruiting
Multiomics study in adults with phenylketonuria
This study looks at adults with phenylketonuria (PKU) using advanced testing (called multiomics) to understand how the condition affects the body. If you took part in a previous PKU study (ECOPHEN) and are willing to give a saliva sample for DNA analysis, this trial may be for you.
AngersAges 18 years+ - NCT07551921Recruiting
Blood Test Validation for Phenylketonuria Monitoring
This study tests a new fingerstick blood test (PheCheck™) designed to help monitor phenylketonuria (PKU), a rare condition affecting how the body processes a protein called phenylalanine. If you or your child has PKU and are already being monitored for it, this study may help improve how your care team tracks the condition.
Chicago, IllinoisAges 1 year+ - NCT07728032Recruiting
How diet and medicine affect gut health in PKU children
This study looks at how a phenylalanine-restricted diet or certain PKU medicines affect gut health in children with PKU. It also includes healthy siblings or household members as a comparison group.
Birmingham, BirminghamAges 3–17 - NCT06332807RecruitingPhase 1/Phase 2
Gene therapy trial for classic PKU
This trial tests a one-time gene therapy called NGGT002 for adults with classic PKU who cannot take or do not respond well to current treatments. The goal is to see if it can safely lower blood phenylalanine levels.
Orange, CaliforniaAges 18–55 - NCT06941532Recruiting
Study of GMP powder for PKU and tyrosinemia
This trial tests a new powdered protein substitute (GMP) for people with PKU or tyrosinemia. It aims to see if it is a safe and effective option to help manage their condition.
BirminghamAges 3 years+ - NCT07220265Recruiting
PKU carriers: How high phenylalanine affects the brain
This study looks at how carrying one copy of the PKU gene might affect thinking and brain health in adults. It will help researchers understand if mild changes in a substance called phenylalanine can impact memory, focus, and brain scans.
Columbia, MissouriAges 18–60 - NCT06337864Recruiting
Large neutral amino acids for adults with classical PKU
This study tests whether a specific mix of large neutral amino acids (LNAAs) can help adults with classical PKU by lowering phenylalanine levels in the brain. It includes an MRI and PET scan to see how the treatment affects brain activity.
CopenhagenAges 18–50
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Phenylketonuria (PKU) trials by city
Studies with a site in or near these metro areas.
Phenylketonuria (PKU) trials by state
Studies with a site anywhere in these states.
Common questions
- Are there clinical trials for phenylketonuria (pku)?
- Yes. Clin2 currently lists 18 recruiting phenylketonuria (pku) studies from the U.S. registry, each rewritten for real people, not researchers, so you can see what it’s testing and who it’s for.
- How do I know if I qualify for a phenylketonuria (pku) trial?
- Each study lists its eligibility criteria — rules about age, diagnosis, and prior treatments. On every Clin2 trial page we explain these in words written for real people and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Does it cost anything to join a phenylketonuria (pku) trial?
- Using Clin2 is always free. Many trials cover the cost of the study treatment and related visits; some reimburse travel. The study team explains exactly what’s covered before you decide.
Related conditions
Clin2 helps you find and understand clinical trials and does not provide medical advice. Study data comes from ClinicalTrials.gov. Talk with your doctor about whether a specific trial is right for you.