Phenylketonuria
Clinical trials for Phenylketonuria explained in plain language.
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New PKU drug could loosen strict diet restrictions
Disease control CompletedThis phase 3 study tests a drug called PTC923 (sepiapterin) in 200 people with phenylketonuria (PKU), a genetic condition that requires a strict low-protein diet. The main goal is to see if the drug is safe over the long term and whether it allows people to eat more protein witho…
Matched conditions: PHENYLKETONURIA
Phase 3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Sep 11, 2026 00:00 UTC
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New pill for PKU shows promise in early human testing
Disease control CompletedThis early-stage study tested an experimental oral drug called JNT-517 in healthy volunteers and people with phenylketonuria (PKU), a genetic condition that causes harmful buildup of the amino acid phenylalanine. The main goals were to check safety and how the body processes the …
Matched conditions: PHENYLKETONURIA
Phase 1/2 • Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. • Aim: Disease control
Last updated Jul 18, 2026 00:00 UTC
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Could a probiotic pill help kids with PKU?
Disease control CompletedThis study tested an engineered probiotic called CBT102-A in 15 children with phenylketonuria (PKU), a condition where the body cannot break down an amino acid called phenylalanine. The children took the probiotic or a placebo for 20 days. The goal was to see if the probiotic cou…
Matched conditions: PHENYLKETONURIA
Early phase 1 • Sponsor: Children's Hospital of Fudan University • Aim: Disease control
Last updated Jun 27, 2026 09:08 UTC
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What should PKU patients eat before exercise? new study aims to find out
Knowledge-focused CompletedThis study looks at how different meals before exercise affect amino acid levels in adults with phenylketonuria (PKU). Ten participants will try water, carbohydrates, or protein before doing aerobic and resistance exercises. Researchers will measure changes in blood amino acids t…
Matched conditions: PHENYLKETONURIA
Sponsor: Hacettepe University • Aim: Knowledge-focused
Last updated Sep 18, 2026 00:00 UTC
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PKU patients may have hidden inflammation, study suggests
Knowledge-focused CompletedThis study looked at 40 adults—some with phenylketonuria (PKU) and some healthy—to see if PKU causes low-level inflammation in the body. Researchers measured inflammatory markers in blood samples. The goal was to better understand how PKU affects overall health, not to test a new…
Matched conditions: PHENYLKETONURIA
Sponsor: University Hospital, Tours • Aim: Knowledge-focused
Last updated Jun 27, 2026 08:06 UTC