Clinical trials
Phenylketonuria clinical trials
Below are recruiting phenylketonuria clinical trials, each written for real people, not researchers. We’re tracking 31 recruiting studies, each written for real people, not researchers, below.
Recruiting studies
- 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 - 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 - NCT06687733RecruitingPhase 1/Phase 2
Gene therapy for adults with classic PKU
This trial tests a new gene therapy called NGGT002 for adults with classic PKU (cPKU) whose phenylalanine levels are not well controlled. The goal is to see if it can safely lower blood Phe levels and reduce the need for a strict diet.
Bengbu, AnhuiAges 18–55 - NCT07685210RecruitingPhase 1
GenSci144 tablet safety study for healthy volunteers
This trial tests the safety of a new tablet called GenSci144 in healthy adults. It helps doctors see if the drug is safe and how the body handles it.
Jinan, ShandongAges 18–45 - 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+ - NCT07477691RecruitingPhase 4
Immune therapy during Palynziq treatment for PKU
This study tests whether combining a drug called methotrexate with Palynziq (a treatment for PKU) can help control immune responses and improve treatment outcomes. It may be an option for adults with PKU who have never used Palynziq or who have not reached target blood levels after at least 24 weeks.
Los Angeles, CaliforniaAges 18–65 - NCT04348708Enrolling by invitation
Long-term follow-up after receiving HMI 102
This study checks how people are doing long-term after they previously received a treatment called HMI 102. It may help researchers understand long-term safety and outcomes so future patients can be better supported.
Boston, MassachusettsAges 18–55
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Common questions
- Are there clinical trials for phenylketonuria?
- Yes. Clin2 currently lists 31 recruiting phenylketonuria 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 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 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.