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Phenylketonuria clinical trials recruiting in the U.S.
14 studies for Phenylketonuria are recruiting participants at sites across the United States.
In phenylketonuria (PKU), your body can't process phenylalanine, an amino acid in most foods. Read about genetics, screening, and special diets.
Cities with the most studies
- Dallas, TX7 studies
- Pittsburgh, PA6 studies
- Indianapolis, IN6 studies
- Portland, OR5 studies
- Chicago, IL5 studies
- Salt Lake City, UT4 studies
- Atlanta, GA4 studies
- Tampa, FL3 studies
- Boston, MA3 studies
- Nashville, TN3 studies
- Milwaukee, WI3 studies
- Aurora, CO3 studies
By state
- California4 studies
- Colorado3 studies
- Florida4 studies
- Georgia4 studies
- Illinois5 studies
- Indiana6 studies
- Massachusetts3 studies
- Oregon5 studies
- Pennsylvania8 studies
- Tennessee4 studies
- Texas7 studies
- Utah4 studies
- Wisconsin3 studies
Recently updated studies
A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria
RecruitingPhase 1
Ages 18 to 69 · All sexes · Updated Sep 28, 2026 · NCT07241234A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
RecruitingPhase 3
Up to age 9 · All sexes · Updated Aug 28, 2026 · NCT06302348PheCheck™ Validation Study
RecruitingObservational study
Ages 1 and older · All sexes · Updated Aug 25, 2026 · NCT07551921A Study of MZE782 in Adults With PKU
RecruitingPhase 2
Ages 18 to 75 · All sexes · Updated Aug 20, 2026 · NCT07694440AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
RecruitingPhase 1/2
Ages 18 to 55 · All sexes · Updated Aug 18, 2026 · NCT06332807A Trial to Examine if Repinatrabit is Processed Differently in Adults With Reduced Liver or Kidney Function Compared to Adults With Normal Liver and Kidney Function
RecruitingPhase 1Healthy volunteers welcome
Ages 18 to 65 · All sexes · Updated Aug 12, 2026 · NCT07713758A Study of JNT-517 in Participants With Phenylketonuria (PKU)
RecruitingPhase 3
Ages 18 and older · All sexes · Updated Jul 8, 2026 · NCT06971731Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
RecruitingPhase 4
Ages 18 to 65 · All sexes · Updated Jun 17, 2026 · NCT07477691Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With Phenylketonuria
RecruitingHealthy volunteers welcome
Ages 11 and older · Women · Updated May 7, 2026 · NCT01659749A Long-Term Study of JNT-517 in Participants With Phenylketonuria
RecruitingPhase 3
Ages 4 and older · All sexes · Updated May 1, 2026 · NCT06628128A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
RecruitingObservational study
All ages · All sexes · Updated Mar 25, 2026 · NCT05813678A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
RecruitingObservational study
All ages · All sexes · Updated Mar 24, 2026 · NCT06305234Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
RecruitingObservational study
Ages 16 and older · All sexes · Updated Jan 14, 2026 · NCT04404530Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria
Recruiting
Ages 10 to 17 · All sexes · Updated Dec 23, 2025 · NCT06560736From ClinicalTrials.gov, data retrieved Sep 30, 2026. Each study sets its own eligibility; the study team decides who can join.