Phenylketonuria



Body Composition, Energy Intake and Expenditure in People With Phenylketonuria


Condition:   Phenylketonuria (PKU)
Intervention:   Other: No intervention
Sponsor:   University of Glasgow
Recruiting


Evaluating the Efficacy of PKU Synergy in Patients Expressing Phenylketonuria or Hyperphenylalaninemia


Conditions:   Phenylketonurias;   Hyperphenylalaninaemia, Type I
Interventions:   Dietary Supplement: Synergy;   Other: Routine
Sponsor:   Nutricia UK Ltd
Enrolling by invitation


Evaluation of PKU Explore


Conditions:   Phenylketonuria;   Inborn Errors of Metabolism
Intervention:   Dietary Supplement: PKU Explore
Sponsors:   Vitaflo International, Ltd;   Birmingham Women's and Children's NHS Foundation Trust
Recruiting


MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria


Condition:   Phenylketonuria
Intervention:  
Sponsors:   Boston Children’s Hospital;   Brigham and Women's Hospital
Recruiting


Evaluation of PKU Start


Conditions:   Phenylketonuria;   Inborn Errors of Metabolism
Intervention:   Dietary Supplement: PKU Start
Sponsor:   Vitaflo International, Ltd
Completed


Simplified Diet Approach in Phenylketonuria


Condition:   Phenylketonuria
Intervention:   Behavioral: Dietary Protein Counting and Free Fruits and Vegetables
Sponsor:   University of Nebraska
Completed


A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302


Condition:   Phenylketonuria
Intervention:   Other: Administration of CANTAB and Subject Global Assessment
Sponsor:   BioMarin Pharmaceutical
Active, not recruiting


PKU Kuvan Imaging Study


Condition:   Phenylketonuria
Intervention:  
Sponsors:   Boston Children’s Hospital;   Beth Israel Deaconess Medical Center;   Brigham and Women's Hospital
Active, not recruiting


Observational Study of Endothelial Dysfunction in Phenylketonuria


Conditions:   Phenylketonuria;   Endothelial Dysfunction
Intervention:  
Sponsors:   Charite University, Berlin, Germany;   Johannes Gutenberg University Mainz
Completed


Simple Breath Test to Examine Phenylalanine Metabolism


Condition:   Phenylketonuria (PKU)
Intervention:  
Sponsors:   University of British Columbia;   Rare Disease Foundation
Completed


Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU


Condition:   Phenylketonuria (PKU)
Interventions:   Drug: BMN165 20mg/day;   Drug: BMN165 40mg/day;   Drug: Placebo
Sponsor:   BioMarin Pharmaceutical
Active, not recruiting


An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165


Condition:   Phenylketonuria
Intervention:   Drug: BMN 165
Sponsor:   BioMarin Pharmaceutical
Completed


Study of a National Cohort of Adult Patients With Phenylketonuria


Conditions:   PKU;   Hyperphenylalaninemia
Intervention:  
Sponsors:   University Hospital, Tours;   Institut National de la Santé Et de la Recherche Médicale, France
Active, not recruiting


Effects of Kuvan on Melatonin Secretion


Condition:   Phenylketonuria (PKU)
Interventions:   Drug: Kuvan;   Dietary Supplement: Large Neutral Amino Acid Therapy
Sponsors:   University of Southern California;   BioMarin Pharmaceutical
Completed


A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks


Condition:   Phenylketonuria
Intervention:   Biological: rAvPAL-PEG
Sponsor:   BioMarin Pharmaceutical
Completed


Liver Cell Transplant for Phenylketonuria


Condition:   Phenylketonuria
Interventions:   Radiation: Preparative Radiation Therapy;   Procedure: Hepatocyte Transplant;   Drug: Immunosuppression;   Other: Liver Evaluation;   Behavioral: Psychological Assessment
Sponsor:   University of Pittsburgh
Suspended


Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria


Condition:   Phenylketonuria
Interventions:   Other: GMP Diet/GMP Medical Foods;   Other: AA Diet/AA Medical Foods
Sponsors:   University of Wisconsin, Madison;   Boston Children’s Hospital
Completed


Kuvan® in Phenylketonuria Patients Less Than 4 Years Old


Condition:   Phenylketonuria
Interventions:   Drug: Kuvan®;   Other: Phenylalanine (Phe)-restricted diet
Sponsor:   BioMarin Pharmaceutical
Completed


Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU)


Condition:   Phenylketonuria
Intervention:   Drug: rAvPAL-PEG
Sponsor:   BioMarin Pharmaceutical
Completed


The Effects of Kuvan on Functional Brain Connectivity in Individuals With Phenylketonuria (PKU)


Condition:   Phenylketonuria
Intervention:   Drug: Sapropterin
Sponsors:   University of Missouri-Columbia;   BioMarin Pharmaceutical
Completed


Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)


Condition:   Phenylketonuria
Intervention:   Drug: rAvPAL-PEG
Sponsor:   BioMarin Pharmaceutical
Active, not recruiting


Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU


Condition:   Phenylketonuria
Intervention:   Drug: rAvPAL-PEG
Sponsor:   BioMarin Pharmaceutical
Completed


Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU


Condition:   Phenylketonuria
Intervention:   Drug: sapropterin dihydrochloride
Sponsor:   BioMarin Pharmaceutical
Active, not recruiting


PKUDOS: Phenylketonuria (PKU) Demographic, Outcomes, and Safety Registry


Conditions:   Phenylketonuria;   Hyperphenylalaninaemia
Intervention:   Drug: Kuvan
Sponsor:   BioMarin Pharmaceutical
Recruiting


Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria


Condition:   Phenylketonuria
Intervention:   Drug: rAvPAL-PEG
Sponsor:   BioMarin Pharmaceutical
Completed

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