Clinical trials · Pittsburgh, PA
Phenylketonuria clinical trials in Pittsburgh
We’re tracking 6 recruiting phenylketonuria studies with a site in or near Pittsburgh — including Phase 1, Phase 1/Phase 2 and Phase 3 studies, each written for real people, not researchers.
Recruiting studies near Pittsburgh
- 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.
Pittsburgh, PennsylvaniaAges 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.
Pittsburgh, PennsylvaniaAges 4 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.
Pittsburgh, PennsylvaniaAges Any age - NCT07241234RecruitingPhase 1
Study of an experimental drug for phenylketonuria (PKU)
This trial tests an investigational drug (AG-181) for people with PKU who have a specific genetic change (R408W mutation). It aims to check if the drug is safe and how it works in your body.
Pittsburgh, PennsylvaniaAges 18–69 - 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.
Pittsburgh, PennsylvaniaAges 18 years+ - 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.
Pittsburgh, PennsylvaniaAges 18–55
Hear when a new Phenylketonuria trial opens near Pittsburgh, PA
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Phenylketonuria trials in other cities
Where these studies are running near Pittsburgh
Institutions with a site for the recruiting phenylketonuria studies listed above.
- University of Pittsburgh Medical Center · 3 studies
- UPMC Children's Hospital of Pittsburgh · 3 studies
What taking part in a phenylketonuria study involves
A screening visit first
Before anything else, the study team checks whether you fit — usually a visit with some tests. You can stop at any point, and screening is typically free.
Care at a nearby site
Study visits happen at a clinic or hospital taking part. Many studies cover the cost of the study treatment and related visits, and some reimburse travel.
You stay in control
Taking part is voluntary and you can leave a study at any time, for any reason, without affecting your regular care.
The team decides eligibility
Our fit check is a helpful first read, not a decision. The study team makes the final call after reviewing your health history.
Looking wider? See all recruiting phenylketonuria studies in Pennsylvania.
Common questions
- Are there phenylketonuria clinical trials near Pittsburgh?
- Yes. We're currently tracking 6 recruiting phenylketonuria studies with a site near Pittsburgh, each rewritten in plain language so you can see what it's testing and who it's for.
- How do I find out if I qualify for a phenylketonuria study near Pittsburgh?
- Each study lists its eligibility rules — age, diagnosis, prior treatments. On every trial page we explain these in plain language and offer a short, optional pre-screen for a fit read. The study team makes the final decision.
- Do I have to live in Pittsburgh to take part?
- Not necessarily. These studies have a site near Pittsburgh, but eligibility is about your health, not your address — some people travel to take part, and a few studies reimburse travel. The study team can tell you what's required.
- Does it cost anything to join a phenylketonuria trial?
- Using Clin2 is always free. Many trials cover the study treatment and related visits; some reimburse travel. The study team explains exactly what's covered before you decide.
Not the right time?
New phenylketonuria studies open near Pittsburgh regularly. Set up a health profile and we’ll quietly watch for studies that fit you and email you when one opens.