Not applicableEnded earlyNCT00827762What this trial is testingBehavioral Effects of Kuvan in Children With Mild PhenylketonuriaWho this might be right forPhenylketonuria Washington University School of Medicine 2
Not applicableStudy completedNCT05096988What this trial is testingEvaluation of PKU Sphere LiquidWho this might be right forPhenylketonurias Vitaflo International, Ltd 19
Not applicableLooking for participantsNCT05579548What this trial is testingA Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and BreastfeedingWho this might be right forPhenylketonuria, Maternal BioMarin Pharmaceutical 50
Not applicableStudy completedNCT05995717What this trial is testingEvaluation of PKU UPWho this might be right forPKU Vitaflo International, Ltd 16
Not applicableEnded earlyNCT04210206What this trial is testingShort-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With PhenylketonuriaWho this might be right forPhenylketonurias Klaus Parhofer 13
Testing effectiveness (Phase 2)Study completedNCT00355264What this trial is testingSafety and Efficacy Study of Phenoptin in Subjects With Hyperphenylalaninemia Due to BH4 DeficiencyWho this might be right forTetrahydrobiopterin DeficienciesHyperphenylalaninemia, Non-Phenylketonuric BioMarin Pharmaceutical 12
Not applicableLooking for participantsNCT01659749What this trial is testingEducational, Social Support, and Nutritional Interventions and Their Cumulative Effect on Pregnancy Outcomes and Quality of Life in Teen and Adult Women With PhenylketonuriaWho this might be right forPregnancyPhenylketonuria Emory University 200
Testing effectiveness (Phase 2)Study completedNCT01560286What this trial is testingEvaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 WeeksWho this might be right forPhenylketonuria BioMarin Pharmaceutical 24
Not applicableStudy completedNCT06694662What this trial is testingPKU EASY Microtabs Plus - Acceptability and ToleranceWho this might be right forPhenylketonuria (PKU) Galen Limited 10
Not applicableStudy completedNCT00728676What this trial is testingStudy to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive BehaviorsWho this might be right forPhenylketonuria Shoji Yano 10
Early research (Phase 1)Ended earlyNCT00244218What this trial is testingResponse to Phenylketonuria to Tetrahydrobiopterin (BH4)Who this might be right forPhenylketonuria The University of Texas Medical Branch, Galveston 57
Testing effectiveness (Phase 2)Active Not RecruitingNCT05972629What this trial is testingA Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With PhenylketonuriaWho this might be right forPhenylketonuria Sanofi 32
Large-scale testing (Phase 3)Looking for participantsNCT06971731What this trial is testingJNT-517 in Participants With Phenylketonuria (PKU)Who this might be right forPhenylketonuria Otsuka Pharmaceutical Development & Commercialization, Inc. 120
Not applicableLooking for participantsNCT06941532What this trial is testingGMP Powdered Substitutes in PKU and TYRWho this might be right forPhenylketonuriaTyrosinemia Nutricia UK Ltd 40
Testing effectiveness (Phase 2)Ended earlyNCT03952156What this trial is testingGene Therapy Clinical Study in Adult PKUWho this might be right forPhenylketonuriasPAH Deficiency Homology Medicines, Inc 10
Not applicableStudy completedNCT03167697What this trial is testingEvaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or HyperphenylalaninemiaWho this might be right forPhenylketonuriasHyperphenylalaninaemia, Type I Nutricia UK Ltd 14
Early research (Phase 1)Looking for participantsNCT07685210What this trial is testingGenSci144 Tablets Phase I Clinical TrialWho this might be right forPhenylketonuria Changchun GeneScience Pharmaceutical Co., Ltd. 48
Not applicableWithdrawnNCT03333720What this trial is testingCompliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of PhenylketonuriaWho this might be right forPhenylketonurias Nutricia UK Ltd
Early research (Phase 1)Active Not RecruitingNCT07255599What this trial is testingEvaluation of BH4 Responsiveness in Our PKU PatientsWho this might be right forPhenylketonuria (PKU) Sohag University 20
Large-scale testing (Phase 3)Study completedNCT05099640What this trial is testingPTC923 in Participants With PhenylketonuriaWho this might be right forPhenylketonuria PTC Therapeutics 157