Phenylketonuria
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Phase 2 Study of JNT-517 in Adolescent Participants With Phenylketonuria [Phase 2]
This clinical trial update describes a Phase 2 study of JNT-517 in adolescents with phenylketonuria (PKU), sponsored by Otsuka Pharmaceutica…
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria [Phase 1]
A Phase 1 clinical trial to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of AG-181 in subjects with phenylketonuria, …
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: A Study to Evaluate the Long-Term Safety and Efficacy of JNT-517 in Participants With Phenylketonuria [Phase 3]
This clinical trial update describes a Phase 3 study to evaluate the long-term safety and efficacy of JNT-517 in participants with phenylket…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Effect of Different Meal Types Given Before Exercise on Plasma Amino Acid Levels and Metabolic Control Parameters in Classical Phenylketonuria Patients Undergoing Aerobic and Resistance Exercises [NA]
This clinical trial examines how different meal types consumed before exercise affect plasma amino acids and metabolic control in people wit…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq [Phase 4]
A Phase 4 clinical trial updates on rapid desensitization for adults with hypersensitivity reactions to Palynziq (pegvaliase) in phenylketon…
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CLINICAL_TRIALS research · 2026-03-27Clinical Trial Update: A Study of Sepiapterin in Participants With Phenylketonuria (PKU) [Phase 3]
Phase 3 clinical trial investigating sepiapterin for phenylketonuria is currently recruiting participants.
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CLINICAL_TRIALS research · 2026-03-24Clinical Trial Update: A Long Term, Post-marketing Study of Immune Response in Patients Receiving Palynziq Treatment for PKU (PALisade)
A long-term, post-marketing study to evaluate the immune response in patients treated with Palynziq for phenylketonuria, currently recruitin…
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CLINICAL_TRIALS research · 2026-03-24Clinical Trial Update: A Global, Multicenter Study to Assess Maternal, Fetal and Infant Outcomes of Exposure to Palynziq® (Pegvaliase) During Pregnancy and Breastfeeding
Global multicenter clinical trial update evaluating maternal, fetal, and infant outcomes after exposure to Palynziq (pegvaliase) during preg…
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CLINICAL_TRIALS research · 2026-03-20Clinical Trial Update: Multiomics Approach in Adult Patients With Phenylketonuria
This entry describes a planned multiomics study in adult patients with phenylketonuria that is not yet recruiting.
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: Immune Modulation During Palynziq® Treatment in Adults (IMPALA) [Phase 4]
A Phase 4 clinical trial update (IMPALA) evaluating immune modulation during Palynziq treatment in adults with phenylketonuria; the study is…
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CLINICAL_TRIALS research · 2026-03-11Clinical Trial Update: Impact of Phenylalanine Elevations on Brain and Cognition in Adult PKU Carriers [NA]
The trial investigates the impact of elevated phenylalanine levels on brain function and cognition in adult carriers of phenylketonuria. Rec…
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CLINICAL_TRIALS research · 2026-03-04Clinical Trial Update: Safety and Efficacy Study of NGGT002 in PKU Adult Subjects [EARLY_Phase 1]
An early-phase clinical trial evaluating the safety and efficacy of NGGT002 in adults with phenylketonuria is currently recruiting.
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: The Psychosocial Functioning of Adults With Phenylketonuria.
Clinical trial update on the psychosocial functioning of adults with phenylketonuria, sponsored by Central Hospital in Nancy, France; status…
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CLINICAL_TRIALS research · 2026-01-15Clinical Trial Update: PKU Protein Requirements [NA]
Emory University is conducting a clinical trial to study protein requirements in phenylketonuria, but recruitment has not yet started.
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CLINICAL_TRIALS research · 2026-01-14Clinical Trial Update: Nutritional Impacts of Palynziq on Patients With Phenylketonuria (PKU)
A clinical trial update noting ongoing recruitment for a study evaluating the nutritional impacts of Palynziq (pegvaliase) in people with ph…
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CLINICAL_TRIALS research · 2026-01-06Clinical Trial Update: To Evaluate the Safety and Efficacy of GS1168 Injection in Adult Phenylketonuria [EARLY_Phase 1]
This clinical trial update announces an early Phase 1 study to evaluate the safety and potential efficacy of GS1168 Injection in adults with…
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CLINICAL_TRIALS research · 2025-12-23Clinical Trial Update: Development of Novel Psychological Assessment Tools and Anxiety Intervention for Phenylketonuria [NA]
A University of Missouri-Columbia-recruited study aims to develop novel psychological assessment tools and an anxiety intervention for indiv…
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CLINICAL_TRIALS research · 2025-12-19Clinical Trial Update: GMP Powdered Substitutes in PKU and TYR [NA]
A Nutricia UK Ltd-sponsored clinical trial evaluating GMP powdered substitutes for phenylketonuria and tyrosinemia is currently recruiting.
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CLINICAL_TRIALS research · 2025-12-02Clinical Trial Update: AAV Gene Therapy Clinical Study in Adult Classic PKU (PHEdom) [Phase 1, Phase 2]
An adeno-associated virus (AAV) gene therapy study for adults with classic phenylketonuria (PKU) is in Phase 1/2 and currently recruiting.
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CLINICAL_TRIALS research · 2025-12-01Clinical Trial Update: Evaluation of BH4 Responsiveness in Our PKU Patients [Phase 1]
Phase 1 clinical trial evaluating BH4 (tetrahydrobiopterin) responsiveness in phenylketonuria patients; the study is currently active but no…
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CLINICAL_TRIALS research · 2025-09-23Clinical Trial Update: A 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 Phenylketonuria [Phase 1, Phase 2]
This clinical trial update describes a Phase 1/Phase 2 open-label study to evaluate the safety, tolerability, and efficacy of a single intra…
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CLINICAL_TRIALS research · 2025-07-31Clinical Trial Update: First-in-Human, Multiple Part Clinical Study of JNT-517 in Healthy Participants and in Participants With Phenylketonuria [Phase 1, Phase 2]
This clinical trial update reports a first-in-human, multi-part Phase 1/2 study of JNT-517 in healthy participants and in individuals with p…
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CLINICAL_TRIALS research · 2025-05-15Clinical Trial Update: Phe for Me? The Effects of L-Phe on PKU Carriers and Non-carriers [NA]
This University of Guelph study investigates the effects of the amino acid L-phenylalanine on individuals who are carriers of phenylketonuri…
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CLINICAL_TRIALS research · 2025-05-13Clinical Trial Update: Brain Aging in Phenylketonuria
A recruiting clinical trial is investigating brain aging in phenylketonuria (PKU). Sponsored by Insel Gruppe AG and University Hospital Bern…
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CLINICAL_TRIALS research · 2025-04-23Clinical Trial Update: A Self-test Home-use Blood Phenylalanine Monitoring System Under the Brand Name Egoo Phe System Has Been Developed for Measurement of Phenylalanine (Phe) in Individuals Diagnosed With Phenylketonuria (PKU): The Study Purpose is to Evaluate Accuracy and Usability
A clinical trial is evaluating the accuracy and usability of the home-use Egoo Phe System for measuring phenylalanine levels in people with …