PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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Tiny tablets could make PKU management easier for patients
Disease control Not yet recruitingThis study tests a new microtablet protein substitute for people with phenylketonuria (PKU), a condition requiring a strict low-protein diet. The microtablets combine protein with vitamins and minerals, aiming to be easier to take than current liquid or powder options. About 20 a…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: NA • Sponsor: Nutricia UK Ltd • Aim: Disease control
Last updated Jun 28, 2026 00:00 UTC
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One-Shot gene therapy could free PKU patients from strict diet
Disease control Not yet recruitingThis early-phase trial tests a single injection of a gene therapy called GS1168 in 9 adults with PKU whose condition is not well controlled by diet or other treatments. The therapy uses a harmless virus to deliver a working copy of the PAH gene, which is faulty in PKU, to help th…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: EARLY_PHASE1 • Sponsor: Gritgen Therapeutics Co., Ltd. • Aim: Disease control
Last updated Jun 27, 2026 08:08 UTC
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PKU mystery: why do some patients thrive despite high phe levels?
Knowledge-focused Not yet recruitingThis study looks at how blood phenylalanine (Phe) levels affect thinking, emotions, and daily functioning in adults with PKU. Researchers will ask 30 adults with PKU and their relatives to complete questionnaires. The goal is to understand why some people with high Phe levels hav…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: Central Hospital, Nancy, France • Aim: Knowledge-focused
Last updated Jun 27, 2026 09:05 UTC