Phenylketonuria
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CLINICAL_TRIALS trial_result · 2021-10-12Clinical Trial Update: Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003) [Phase 2]
Phase 2 long-term extension trial update for rAvPAL-PEG in phenylketonuria (PKU) completed.
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CLINICAL_TRIALS trial_result · 2021-05-13Clinical Trial Update: Safety and Tolerability of SYNB1618 in Healthy Adult Volunteers and Adult Subjects With Phenylketonuria [Phase 1, Phase 2]
Phase 1/2 clinical trial evaluating safety and tolerability of SYNB1618 in healthy adults and adults with phenylketonuria has completed.
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CLINICAL_TRIALS trial_result · 2020-12-22Clinical Trial Update: Effect of Kuvan on Neurocognitive Function, Blood Phenylalanine Level, Safety, and Pharmacokinetics in Children With PKU [Phase 3]
A Phase 3 trial evaluating Kuvan (tetrahydrobiopterin) effects on neurocognitive function, phenylalanine levels, safety, and pharmacokinetic…
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CLINICAL_TRIALS trial_result · 2020-06-04Clinical Trial Update: A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
Phase 3 substudy assessing executive function in adults with phenylketonuria participating in the main Phase 3 trial has completed; results …
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CLINICAL_TRIALS trial_result · 2020-01-13Clinical Trial Update: BH4 Responsiveness in PAH Deficiency PKU Patients [Phase 4]
Phase 4 clinical trial update on BH4 responsiveness in phenylketonuria patients with PAH deficiency.
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CLINICAL_TRIALS trial_result · 2019-10-08Clinical Trial Update: Kuvan Therapy in Phenylketonuria (PKU): The Effect of Blood Phenylalanine Concentration on Kuvan Response [Phase 2]
Phase 2 trial update evaluating how blood phenylalanine levels influence Kuvan response in phenylketonuria.
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CLINICAL_TRIALS trial_result · 2019-07-12Clinical Trial Update: Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU [Phase 3]
Phase 3 trial evaluating self-administered injections of BMN165 for adults with phenylketonuria has completed; results were posted in 2019.
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CLINICAL_TRIALS trial_result · 2019-02-27Clinical Trial Update: A Study to Evaluate Subcutaneously Administered rAvPAL-PEG in Patients With Phenylketonuria for 24 Weeks [Phase 2]
Phase 2 clinical trial evaluating subcutaneously administered rAvPAL-PEG in phenylketonuria has completed, with results posted.
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CLINICAL_TRIALS trial_result · 2019-02-26Clinical Trial Update: Safety, Tolerability, and Efficacy Study of rAvPAL-PEG Administered Daily in Subjects With Phenylketonuria (PKU) [Phase 2]
Phase 2 trial update on rAvPAL-PEG for phenylketonuria assessing safety, tolerability, and efficacy.
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CLINICAL_TRIALS trial_result · 2019-02-26Clinical Trial Update: Dose-Finding Study to Evaluate the Safety, Efficacy, & Tolerability of Multiple Doses of rAvPAL-PEG in Subjects With PKU [Phase 2]
Phase 2 dose-finding trial evaluating safety, efficacy, and tolerability of multiple doses of rAvPAL-PEG in phenylketonuria. Results posted …
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CLINICAL_TRIALS trial_result · 2018-09-10Clinical Trial Update: An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 [Phase 3]
Open-label Phase 3 trial of BMN 165 in adults with phenylketonuria who were not previously treated with BMN 165 has completed and results we…
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CLINICAL_TRIALS trial_result · 2018-06-18Clinical Trial Update: Sapropterin in Individuals With Phenylketonuria
This clinical trial update reports that a study evaluating sapropterin for phenylketonuria has completed.
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CLINICAL_TRIALS trial_result · 2017-03-03Clinical Trial Update: Phase 2 Study of Glycomacropeptide Versus Amino Acid Diet for Management of Phenylketonuria [NA]
Phase 2 study comparing glycomacropeptide to amino acid diet for phenylketonuria has completed with results posted in 2017.
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CLINICAL_TRIALS trial_result · 2017-02-23Clinical Trial Update: Observational Study of Endothelial Dysfunction in Phenylketonuria
This observational trial evaluating endothelial dysfunction in individuals with phenylketonuria has completed.
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CLINICAL_TRIALS trial_result · 2017-02-01Clinical Trial Update: Safety and Tolerability Study of rAvPAL-PEG to Treat Phenylketonuria [Phase 1]
This document reports the completion of a Phase 1 safety and tolerability study of rAvPAL-PEG for phenylketonuria.
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CLINICAL_TRIALS trial_result · 2017-01-16Clinical Trial Update: Antioxidant Signature in Adult Patients With Phenylketonuria
A completed clinical trial investigated antioxidant signatures in adult patients with phenylketonuria.
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CLINICAL_TRIALS trial_result · 2016-11-18Clinical Trial Update: Effects of Kuvan on Melatonin Secretion [Phase 4]
Phase 4 trial evaluating the effect of Kuvan (sapropterin dihydrochloride) on melatonin secretion in individuals with phenylketonuria has be…
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CLINICAL_TRIALS trial_result · 2016-05-23Clinical Trial Update: Impact of Phenylketonuria-type Diet on Appetite, Appetite Hormones and Diet Induced Thermogenesis [NA]
A completed clinical trial from the University of Glasgow examining how a phenylketonuria-type diet affects appetite, appetite hormones, and…
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CLINICAL_TRIALS trial_result · 2016-05-12Clinical Trial Update: Kuvan® in Phenylketonuria Patients Less Than 4 Years Old [Phase 3]
Phase 3 trial update for Kuvan in phenylketonuria patients under 4 years old; study completed with results posted in 2016.
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CLINICAL_TRIALS trial_result · 2016-04-20Clinical Trial Update: A Pilot Study on Diurnal Variation [NA]
This clinical trial update summarizes results from a terminated pilot study investigating diurnal variation in Phenylketonuria (PKU).
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CLINICAL_TRIALS trial_result · 2016-02-17Clinical Trial Update: Neurocognitive Outcomes in Mild Hyperphenylalaninemia (MHP)MHP Study
A completed clinical trial update evaluating neurocognitive outcomes in individuals with mild hyperphenylalaninemia/phenylketonuria; the sni…
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CLINICAL_TRIALS trial_result · 2016-02-12Clinical Trial Update: Sapropterin on Cognitive Abilities in Young Adults With Phenylketonuria [Phase 2]
A Phase 2 trial evaluating sapropterin for cognitive abilities in young adults with phenylketonuria was terminated with results posted in 20…
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CLINICAL_TRIALS trial_result · 2016-02-08Clinical Trial Update: Phenylketonuria and Hyperphenylalaninemia Nutrition Study
This entry reports the completion of a nutrition-focused clinical trial studying phenylketonuria and hyperphenylalaninemia. The provided sni…
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CLINICAL_TRIALS trial_result · 2016-02-01Clinical Trial Update: Safety and Therapeutic Effects of Sapropterin Dihydrochloride on Neuropsychiatric Symptoms in Phenylketonuria (PKU) Patients [Phase 3]
Phase 3 study evaluating the safety and potential neuropsychiatric benefits of sapropterin dihydrochloride in phenylketonuria patients; the …
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CLINICAL_TRIALS trial_result · 2015-12-11Clinical Trial Update: Biological Variation of Phenylalanine in Patients With Hyperphenylalaninemia
A completed clinical trial examining the biological variation of phenylalanine levels in patients with hyperphenylalaninemia/phenylketonuria…