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Condition: Phenylketonuria Pku Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Phenylketonuria Pku

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
Phase N/A · 11 location(s) · BioMarin Pharmaceutical · NCT06305234A Study of JNT-517 in Participants With Phenylketonuria (PKU)
Phase 3 · 26 location(s) · Otsuka Pharmaceutical Development & Commercialization, Inc. · NCT06971731Safety and Efficacy Study of NGGT002 in PKU Adult Subjects
EARLY_Phase 1 · 1 location(s) · The First Affiliated Hospital of Bengbu Medical University · NCT06061614Impact Of A Phe-Restricted Diet On Gut Health In Children With PKU
Phase N/A · 1 location(s) · Birmingham Women's and Children's NHS Foundation Trust · NCT07728032Walking Program in Fatty Liver Children With Phenylketonuria
NA · 1 location(s) · Cairo University · NCT06718842A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During P
Phase N/A · 4 location(s) · BioMarin Pharmaceutical · NCT05579548A Long-Term Study of JNT-517 in Participants With Phenylketonuria
Phase 3 · 12 location(s) · Otsuka Pharmaceutical Development & Commercialization, Inc. · NCT06628128GMP Powdered Substitutes in PKU and TYR
NA · 1 location(s) · Nutricia UK Ltd · NCT06941532A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonu
Phase 1 · 4 location(s) · Agios Pharmaceuticals, Inc. · NCT07241234A Trial to Examine the Interaction of Repinatrabit With Ethinyl Estradiol/Norethindrone, Metformin,Carbamazepine, Rosuva
Phase 1 · 1 location(s) · Otsuka Pharmaceutical Development & Commercialization, Inc. · NCT07446400AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom)
Phase 1/Phase 2 · 5 location(s) · NGGT INC. · NCT06332807A Study of MZE782 in Adults With PKU
Phase 2 · 2 location(s) · Maze Therapeutics · NCT07694440A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
Phase N/A · 26 location(s) · BioMarin Pharmaceutical · NCT05813678Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers
NA · 1 location(s) · University of Missouri-Columbia · NCT07220265Brain Aging in Phenylketonuria
Phase N/A · 1 location(s) · Insel Gruppe AG, University Hospital Bern · NCT06969209Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
Phase N/A · 2 location(s) · Emory University · NCT04404530Multiomics Approach in Adult Patients With Phenylketonuria
Phase N/A · 15 location(s) · University Hospital, Tours · NCT07484945Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in Cla
NA · 1 location(s) · Hacettepe University · NCT07526909Announcement of Rare Metabolic Diseases in Systematic Newborn Screening: the Phenylketonuria Experience.
Phase N/A · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT06289348Eating Disorders in Patients With Phenylketonuria
Phase N/A · 1 location(s) · Central Hospital, Nancy, France · NCT07698743Immune Modulation During Palynziq® Treatment in Adults (IMPALA)
Phase 4 · 12 location(s) · BioMarin Pharmaceutical · NCT07477691Effect of Large Neutral Amino Acids in Adults With Classical Phenylketonuria
NA · 2 location(s) · Rigshospitalet, Denmark · NCT06337864Metabolic Control and Patient Well-being in Phenylketonuria: do Guidelines Make a Difference?
Phase N/A · 1 location(s) · University Children's Hospital, Zurich · NCT05128149GenSci144 Tablets Phase I Clinical Trial
Phase 1 · 1 location(s) · Changchun GeneScience Pharmaceutical Co., Ltd. · NCT07685210A Study of Sepiapterin in Participants With Phenylketonuria (PKU)
Phase 3 · 10 location(s) · PTC Therapeutics · NCT06302348A Clinical Study on the Safety and Tolerability of PL54 Injection in Adult Patients With Phenylketonuria (PKU)
Phase 1 · 1 location(s) · Chongqing Peg-Bio Biopharm Co., Ltd. · NCT07672756Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria
NA · 1 location(s) · University of Missouri-Columbia · NCT06560736Safety and Efficacy Study of NGGT002 in cPKU Adult Subjects
Phase 1/Phase 2 · 2 location(s) · NGGT (Suzhou) Biotechnology Co., Ltd. · NCT06687733Educational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality
NA · 1 location(s) · Emory University · NCT01659749
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.