Phenylketonuria



MRI Spectroscopy and Neuropsychological Functioning in Phenylketonuria


Condition:   Phenylketonuria
Intervention:  
Sponsors:   Boston Children’s Hospital;   Brigham and Women's Hospital
Not yet recruiting - verified March 2017


Evaluation of PKU Start


Conditions:   Phenylketonuria;   Inborn Errors of Metabolism
Intervention:   Dietary Supplement: PKU Start
Sponsor:   Vitaflo International, Ltd
Recruiting - verified April 2017


GMP Drink for PKU Study


Condition:   Phenylketonuria
Intervention:   Dietary Supplement: Glycomacropeptide-based protein substitute
Sponsor:   Nutricia UK Ltd
Recruiting - verified September 2016


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 - verified January 2017


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 - verified April 2017


Antioxidant Signature in Adult Patients With Phenylketonuria


Conditions:   PKU;   Hyperphenylalaninemia
Interventions:   Other: Blood sample;   Other: Urine sample
Sponsors:   University Hospital, Tours;   Institut National de la Santé Et de la Recherche Médicale, France
Completed - verified January 2017


Observational Study of Endothelial Dysfunction in Phenylketonuria


Conditions:   Phenylketonuria;   Endothelial Dysfunction
Intervention:  
Sponsors:   Charite University, Berlin, Germany;   Johannes Gutenberg University Mainz
Completed - verified February 2017


Simple Breath Test to Examine Phenylalanine Metabolism


Condition:   Phenylketonuria (PKU)
Intervention:  
Sponsors:   University of British Columbia;   Rare Disease Foundation
Completed - verified November 2016


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 - verified September 2016


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 - verified January 2017


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 - verified November 2016


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 - verified November 2016


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 - verified February 2017


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 - verified January 2017


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 - verified October 2016


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 - verified January 2017


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 - verified January 2017


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 - verified April 2017


Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria


Condition:   Phenylketonuria
Intervention:   Drug: rAvPAL-PEG
Sponsor:   BioMarin Pharmaceutical
Completed - verified January 2017

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