Phenylketonurias: all 187 studies

Page 2 of 2. Open to participants first (38), then studies with results posted, then the rest; newest registration first within each.

• Evaluation of PKU Explore FranceCompletedNCT04898829Not applicable
• Study of Low-grade Systemic Inflammation in Adult Patients With PhenylketonuriaCompletedNCT04879277Not applicable
• Natural History Clinical Study in Adult PKUTerminatedNCT04768348
• Evaluation of PKU Sphere in ItalyCompletedNCT04679467Not applicable
• Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With PhenylketonuriaCompletedNCT04534842Phase 2
• AAV Gene Therapy Study for Subjects with PKUActive, not recruitingNCT04480567Phase 1/2
• A Prospective Clinical Study of Phenylketonuria (PKU)CompletedNCT04452513
• Life With Phenylketonuria. Adult Neurological Outcome of PCU Screened Patients From 1971 to 2002.TerminatedNCT04433728
• Acceptability and Tolerance of a Ready-to-use Protein Substitute in Tablet Form for the Dietary Management of PhenylketonuriaCompletedNCT04375592
• PKU Skin StrippingCompletedNCT04368624
• Market Research - Acceptability Study for a New PKU Protein SubstituteCompletedNCT04318509Not applicable
• Market Research - Acceptability Trial for a New PKU Amino Acid Based Protein SubstituteCompletedNCT04309331Not applicable
• Early Dietary Treated Patients With Phenylketonuria Can Achieve Normal Growth and Mental Development.Status unknownNCT04302194
• PKU Low Calorie Drink StudyWithdrawnNCT04272736Not applicable
• Safety, Tolerability, Pharmacodynamics and Pharmacokinetics of CDX 6114 in PKU PatientsWithdrawnNCT04256655Phase 1
• A Study Into the Future Cardiovascular Disease (CVD) Risk of Phenylketonuria (PKU) Patients on a Low Phenylalanine Treatment Diet (LPD).Status unknownNCT04251494
• Patient and Observer Reported Outcome Measurements in Inborn Errors of MetabolismCompletedNCT04248062
• BH4 Responsiveness in PAH Deficiency PKU PatientsStatus unknownNCT04227080Phase 4
• Evaluation of PKU Sphere in Maternal PKUCompletedNCT04224142
• Short-term Effects of Good Metabolic Control on Cognitive Function, Wellbeing, and Metabolic Parameters in Adult Patients With PhenylketonuriaTerminatedNCT04210206Not applicable
• Safety and Tolerability of RTX-134 in Adults With PhenylketonuriaTerminatedNCT04110496Phase 1
• PANDA: PKU Amino Acid EvaluationCompletedNCT04086511
• Pharmacodynamics, Safety, Tolerability and Pharmacokinetics of CDX-6114 in Patients With Phenylketonuria (PKU)CompletedNCT04085666Phase 1
• The Effects of CGMP in Children and Adults With PKUCompletedNCT04076176Not applicable
• O2 Transport and Utilization in Health and Lung DiseaseWithdrawnNCT04014712Phase 1
• Gene Therapy Clinical Study in Adult PKUTerminatedNCT03952156Phase 1/2
• Protein Requirements in Adults With Phenylketonuria (PKU)CompletedNCT03939052Not applicable
• Glytactin EfficiEncy in Non or Insufficiently Treated Adult PHENylketonuria PatientsCompletedNCT03924180Not applicable
• Retrospective Observational Safety Effectiveness With Kuvan in hpACompletedNCT03864029
• SNAP: Study Nutrients in Adult PKUCompletedNCT03858101
• Nutrition Status of Adults With PKU Before and During Treatment With PegvaliaseCompletedNCT03856203
• Nutritional Status in PhenylketonuriaCompletedNCT03820804
• Nutrition Status of Adults Treated With PegvaliaseCompletedNCT03792451
• Phenylalanine and Its Impact on CognitionCompletedNCT03788343Phase 4
• Effects of Synergy on Nutrient Intake and Acceptability in Phenylketonuria (PKU)CompletedNCT03777826Not applicable
• A 16 Week Study Evaluating the Introduction of a GMP Based Protein Substitute in Participants With PKUCompletedNCT03771391Not applicable
• Early Check: Expanded Screening in NewbornsActive, not recruitingNCT03655223
• Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With PhenylketonuriaCompletedNCT03516487Phase 1/2
• A Non-Interventional Interview Study of Phenylketonuria (PKU) AdultsCompletedNCT03505125
• Evaluation of PKU SphereCompletedNCT03419819Not applicable
• Compliance, Tolerance and Acceptability of a Tablet Protein Substitute for the Dietary Management of PhenylketonuriaWithdrawnNCT03333720Not applicable
• Body Composition, Energy Intake and Expenditure in People With PhenylketonuriaStatus unknownNCT03309345
• Evaluation of PKU ExploreCompletedNCT03168399Not applicable
• Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or HyperphenylalaninemiaCompletedNCT03167697Not applicable
• MRI Spectroscopy and Neuropsychological Functioning in PhenylketonuriaCompletedNCT03097250
• Evaluation of PKU StartCompletedNCT03058848Not applicable
• GMP Drink for PKU StudyCompletedNCT02915510Not applicable
• Simplified Diet Approach in PhenylketonuriaCompletedNCT02555579Not applicable
• Testing of Four Home Phenylalanine Monitoring Prototype DevicesCompletedNCT02445521
• Impact of Phenylketonuria-type Diet on Appetite, Appetite Hormones and Diet Induced ThermogenesisCompletedNCT02440932Not applicable
• The Brain and Neuropsychological Functioning in Adults With Sapropterin Dihydrochloride Treated PhenylketonuriaCompletedNCT02297347
• Antioxidant Signature in Adult Patients With PhenylketonuriaCompletedNCT02212288
• Observational Study of Endothelial Dysfunction in PhenylketonuriaCompletedNCT02176603
• Simple Breath Test to Examine Phenylalanine MetabolismCompletedNCT02009904
• Kuvan®'s Effect on the Cognition of Children With PhenylketonuriaCompletedNCT01965912Phase 4
• Protein Requirements in Children With Phenylketonuria (PKU)CompletedNCT01965691Not applicable
• Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP StudyCompletedNCT01924026
• Moderate Intensity Exercise and PhenylketonuriaTerminatedNCT01904708Not applicable
• Phenylketonuria and Hyperphenylalaninemia Nutrition StudyCompletedNCT01879995
• Biological Variation of Phenylalanine in Patients With HyperphenylalaninemiaCompletedNCT01869972
• The Effects of Sapropterin Dihydrochloride Supplementation on in Vivo Redox Status in Patients With Classical PKUWithdrawnNCT01650909
• Study of a National Cohort of Adult Patients With PhenylketonuriaCompletedNCT01619722
• Liver Cell Transplant for PhenylketonuriaTerminatedNCT01465100Phase 1/2
• Neuroimaging and Neurocognitive Assessment and Response to Sapropterin Dihydrochloride Treatment in PhenylketonuriaWithdrawnNCT01412437Not applicable
• Evaluation of Behavior, Executive Function, Neurotransmitter Function and Genomic Expression Kuvan NonrespondersCompletedNCT01274026
• Bone Mineral Density in Adults With HyperphenylalaninemiaCompletedNCT01209819
• Follow-up of Adult Phenylketonuria (PKU) PatientsCompletedNCT01096758
• Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 DeficiencyCompletedNCT01016392
• The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)CompletedNCT00964236
• Quantitative Requirements of Docosahexaenoic Acid for Neural Function in Children With PhenylketonuriaCompletedNCT00909012Not applicable
• The Effect of Supplemental Docosahexaenoic Acid (DHA) on Neurocognitive Outcomes in Teen and Adult Women With Phenylketonuria(PKU)CompletedNCT00892554Not applicable
• Behavioral Effects of Kuvan in Children With Mild PhenylketonuriaTerminatedNCT00827762
• A Phase 1 Study to Evaluate Effects of Sapropterin Dihydrochloride on QTc Intervals in Healthy Adult SubjectsCompletedNCT00789568Phase 1
• PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety RegistryCompletedNCT00778206
• Sapropterin in Individuals With PhenylketonuriaCompletedNCT00730080
• Study to Evaluate the Effects of Kuvan on Individuals With Phenylketonuria (PKU) With Maladaptive BehaviorsCompletedNCT00728676
• Safety and Tolerability Study of rAvPAL-PEG to Treat PhenylketonuriaCompletedNCT00634660Phase 1
• Sapropterin Expanded Access ProgramApproved for marketingNCT00484991
• Long-Term Tetrahydrobiopterin Treatment in PKU Patients of 0-18 Years - Study on Phenylalanine Tolerance and SafetyTerminatedNCT00432822Phase 2/3
• Hyperphenylalaninemia in Cerebral MalariaCompletedNCT00338520
• The Early History of Universal Screening for Metabolic DisordersCompletedNCT00309400
• Study of BH4, a New and Simple Treatment of Mild PKUCompletedNCT00260000Phase 2
• Response to Phenylketonuria to Tetrahydrobiopterin (BH4)TerminatedNCT00244218Phase 1
• A Phase 3, Multicenter, Open-Label Extension Study of Phenoptin in Subjects With PKU Who Have Elevated Phenylalanine LevelsCompletedNCT00225615Phase 3
• Study to Evaluate the Response to and Safety of an 8-Day Course of Phenoptin™ Treatment in Subjects With PhenylketonuriaCompletedNCT00104260Phase 2
• Low Phenylalanine Diet for Mothers With Phenylketonuria (PKU)CompletedNCT00065299Not applicable
• Study of a Phenylalanine Restricted Diet During Pregnancy to Prevent Symptoms in Offspring of Patients With PhenylketonuriaCompletedNCT00006142Not applicable

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