PHENYLKETONURIAS
Clinical trials for PHENYLKETONURIAS explained in plain language.
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New PKU nighttime treatment tested for better blood control
Disease control CompletedThis study tested if a new protein substitute called PKU GOLIKE, taken at night, helps keep blood phenylalanine levels more stable in children with PKU compared to their standard amino acid treatment. Sixteen children tried both treatments for one week each, with a break in betwe…
Matched conditions: PHENYLKETONURIAS
Phase: NA • Sponsor: APR Applied Pharma Research s.a. • Aim: Disease control
Last updated Mar 27, 2026 12:37 UTC
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Taste test for Life-Saving medical foods
Disease control CompletedThis study tested whether new medical food products called 'express plus' are acceptable for people with rare inherited metabolic disorders like PKU. Researchers wanted to see if patients found these products tasty, easy to use, and could stick to taking them as part of their spe…
Matched conditions: PHENYLKETONURIAS
Phase: NA • Sponsor: Vitaflo International, Ltd • Aim: Disease control
Last updated Mar 23, 2026 15:18 UTC
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Taste test for tiny patients: can a new spoonable food help manage a rare disorder?
Disease control CompletedThis study tested a new spoonable medical food called PKU Explore for infants and toddlers with phenylketonuria (PKU). Researchers wanted to see if the children would accept the food, tolerate it well, and if it could help control their blood phenylalanine (Phe) levels while supp…
Matched conditions: PHENYLKETONURIAS
Phase: NA • Sponsor: Vitaflo International, Ltd • Aim: Disease control
Last updated Mar 16, 2026 15:27 UTC
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New Finger-Prick test could simplify PKU monitoring
Diagnosis CompletedThis study tested a new device called PheCheck that measures phenylalanine levels from a simple finger-prick of blood. It aimed to see if this quick, at-home test is as accurate as the standard, more complex lab test. The study involved 30 people aged 10 and older who have PKU.
Matched conditions: PHENYLKETONURIAS
Sponsor: Lumos Diagnostics • Aim: Diagnosis
Last updated Mar 30, 2026 14:34 UTC
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Scientists scan brains to see if PKU drug repairs damage
Knowledge-focused CompletedThis study looked at whether Palynziq, a medication that lowers phenylalanine levels in people with PKU, leads to improvements in brain health and thinking skills. Researchers used brain scans and cognitive tests to measure changes in 6 adults with PKU who had been successfully t…
Matched conditions: PHENYLKETONURIAS
Sponsor: University of Missouri-Columbia • Aim: Knowledge-focused
Last updated Mar 30, 2026 14:32 UTC