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Phenylketonuria clinical trials in Pittsburgh, PA

6 studies for Phenylketonuria are looking for participants at sites in and around Pittsburgh (within about 30 miles). Open a study to see who can join and how to contact the study team.

Recruiting for a study here? Feature it at the top of this page — $99 for 30 days →

A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria

RecruitingPhase 1
  • UPMC Children's Hospital of Pittsburgh, Pittsburgh
Ages 18 to 69 · All sexes · Updated Sep 28, 2026 · NCT07241234

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

RecruitingPhase 1/2
  • University of Pittsburgh Medical Center, Pittsburgh
Ages 18 to 55 · All sexes · Updated Aug 18, 2026 · NCT06332807

A Study of JNT-517 in Participants With Phenylketonuria (PKU)

RecruitingPhase 3
  • University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh, Pittsburgh
Ages 18 and older · All sexes · Updated Jul 8, 2026 · NCT06971731

A Long-Term Study of JNT-517 in Participants With Phenylketonuria

RecruitingPhase 3
  • University of Pittsburgh Medical Center (UPMC) - Children's Hospital of Pittsburgh, Pittsburgh
Ages 4 and older · All sexes · Updated May 1, 2026 · NCT06628128

A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)

RecruitingObservational study
  • University of Pittsburgh Medical Center, Pittsburgh
All ages · All sexes · Updated Mar 25, 2026 · NCT05813678

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)

RecruitingObservational study
  • University of Pittsburgh Medical Center, Pittsburgh
All ages · All sexes · Updated Mar 24, 2026 · NCT06305234

About Phenylketonuria

In phenylketonuria (PKU), your body can't process phenylalanine, an amino acid in most foods. Read about genetics, screening, and special diets.

More about Phenylketonuria on MedlinePlus

Phenylketonuria trials in other cities

Other studies in Pittsburgh

From ClinicalTrials.gov, data retrieved Oct 5, 2026. Each study sets its own eligibility; the study team decides who can join.