Phenylketonuria
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CLINICAL_TRIALS trial_result · 2026-06-23Clinical Trial Update: A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria [Phase 3]
Long-term safety study update for PTC923 in participants with Phenylketonuria (Phase 3).
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CLINICAL_TRIALS trial_result · 2026-04-24Clinical Trial Update: A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria [Phase 3]
This entry provides an update on a Phase 3 long-term safety study of PTC923 in people with Phenylketonuria, with the trial currently active …
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CLINICAL_TRIALS trial_result · 2026-04-08Clinical Trial Update: A Study to Evaluate the Safety and Efficacy of JNT-517 in Participants With Phenylketonuria (PKU) [Phase 3]
Phase 3 study evaluating the safety and efficacy of JNT-517 in phenylketonuria is currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-03-25Clinical Trial Update: A Long-term, Post-marketing Safety Study of Palynziq in Patients With PKU (PALace)
This document outlines a long-term, post-marketing safety study of Palynziq in patients with phenylketonuria (PKU), and the study is current…
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CLINICAL_TRIALS trial_result · 2026-03-11Clinical Trial Update: Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU. [NA]
Terminated open-label, randomized 2-way crossover trial assessing daily phenylalanine fluctuation in phenylketonuria patients, comparing PKU…
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CLINICAL_TRIALS trial_result · 2026-02-27Clinical Trial Update: Efficacy and Safety of Orally Administered Engineered Probiotics (CBT102-A) for the Treatment of Children With Phenylketonuria [EARLY_Phase 1]
A Phase 1 trial evaluated the efficacy and safety of orally administered engineered probiotic CBT102-A in children with phenylketonuria; the…
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CLINICAL_TRIALS trial_result · 2026-02-19Clinical Trial Update: Evaluation of PKU UP [NA]
A clinical trial update for PKU UP, sponsored by Vitaflo International, Ltd, indicates the study in phenylketonuria is active but not recrui…
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CLINICAL_TRIALS trial_result · 2025-12-29Clinical Trial Update: Preliminary Study of Brain Effects of Palynziq-Related Changes in Phenylalanine in Individuals With PKU
This completed preliminary clinical trial evaluated how Palynziq-related changes in phenylalanine impact brain function in individuals with …
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CLINICAL_TRIALS trial_result · 2025-12-22Clinical Trial Update: Study of Low-grade Systemic Inflammation in Adult Patients With Phenylketonuria [NA]
This clinical trial update indicates that the study examining low-grade systemic inflammation in adult phenylketonuria patients has complete…
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CLINICAL_TRIALS trial_result · 2025-11-26Clinical Trial Update: PKU EASY Microtabs Plus - Acceptability and Tolerance [NA]
The completed trial update reports on the acceptability and tolerability of PKU EASY Microtabs Plus for phenylketonuria. No additional effic…
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CLINICAL_TRIALS trial_result · 2025-09-15Clinical Trial Update: PheCheck Feasibility Study
Lumos Diagnostics reports that the PheCheck feasibility study for phenylketonuria has completed. The update outlines preliminary findings an…
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CLINICAL_TRIALS trial_result · 2025-09-12Clinical Trial Update: Study to Evaluate the Safety and Efficacy of Pegvaliase in Adolescents (Ages 12-17) With Phenylketonuria [Phase 3]
Phase 3 trial evaluating pegvaliase for phenylketonuria in adolescents (ages 12-17) is currently active but not recruiting.
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CLINICAL_TRIALS trial_result · 2025-06-08Clinical Trial Update: Evaluation of Phe Fluctuation in PKU Pts Treated With PKU GOLIKE Versus Standard Amino Acid Protein Substitute. [NA]
This completed clinical trial compared phenylalanine fluctuations in phenylketonuria patients treated with PKU GOLIKE versus a standard amin…
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CLINICAL_TRIALS trial_result · 2025-04-04Clinical Trial Update: Effect of L-carnitine Supplementation on Phenylalanine and Brain-derived Neurotrophic Factor Levels in Infants and Children With Phenylketonuria [Phase 4]
This Phase 4 clinical trial update describes evaluating L-carnitine supplementation for its effect on phenylalanine and brain-derived neurot…
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CLINICAL_TRIALS trial_result · 2025-03-04Clinical Trial Update: Amino Acid Kinetics of GMP-AA in Healthy Human Volunteers [NA]
The clinical trial evaluated amino acid kinetics of GMP-AA in healthy volunteers. The study has completed.
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CLINICAL_TRIALS trial_result · 2025-01-24Clinical Trial Update: Iron Supplements Effect on b-Phe Levels in PKU Patients During 3 Years Study [Phase 2]
A Phase 2 clinical trial evaluating the effect of iron supplements on blood phenylalanine levels in patients with phenylketonuria has comple…
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CLINICAL_TRIALS trial_result · 2024-12-06Clinical Trial Update: PKU Carriers Trial (Pilot Study): Impact on Cognition, Mental Health, Blood Pressure and Metabolism [NA]
The University of Guelph is conducting a pilot study in genetic PKU carriers to evaluate effects on cognition, mental health, blood pressure…
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CLINICAL_TRIALS trial_result · 2024-10-09Clinical Trial Update: Determining the Most Efficient Screening Cut Off Levels for Phenylketonuria (PKU)
A completed clinical trial from Central Hospital in Nancy, France, evaluated different phenylketonuria newborn screening cutoff levels to de…
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CLINICAL_TRIALS trial_result · 2024-06-20Clinical Trial Update: Efficacy and Safety of SYNB1934 in Patients With PKU (SYNPHENY-3) [Phase 3]
Phase 3 trial SYNPHENY-3 evaluating SYNB1934 in phenylketonuria was terminated. Results were posted in June 2024.
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CLINICAL_TRIALS trial_result · 2024-01-10Clinical Trial Update: A Study of PTC923 in Participants With Phenylketonuria [Phase 3]
Phase 3 study of PTC923 in phenylketonuria has completed; results posted.
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CLINICAL_TRIALS trial_result · 2023-04-24Clinical Trial Update: Kuvan®'s Effect on the Cognition of Children With Phenylketonuria [Phase 4]
Phase 4 trial assessing Kuvan® (tetrahydrobiopterin) effect on cognition in children with phenylketonuria has completed.
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CLINICAL_TRIALS trial_result · 2023-03-07Clinical Trial Update: Phenylalanine and Its Impact on Cognition [Phase 4]
Phase 4 clinical trial update examining phenylalanine and its impact on cognition in phenylketonuria.
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CLINICAL_TRIALS trial_result · 2022-10-17Clinical Trial Update: Efficacy and Safety of SYNB1618 and SYNB1934 in Adult Patients With Phenylketonuria [Phase 2]
Phase 2 trial update on the efficacy and safety of SYNB1618 and SYNB1934 in adults with phenylketonuria has been completed.
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CLINICAL_TRIALS trial_result · 2022-04-27Clinical Trial Update: The Effectiveness of Kuvan in Amish PKU Patients [Phase 4]
Phase 4 trial reporting on the effectiveness of Kuvan in Amish patients with phenylketonuria has been completed with results posted.
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CLINICAL_TRIALS trial_result · 2022-02-17Clinical Trial Update: Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU [Phase 3]
Phase 3 trial evaluating safety and efficacy of self-administered pegvaliase (>40 mg/day) in adults with phenylketonuria (PKU) has completed…