PHENYLKETONURIA
Clinical trials for PHENYLKETONURIA explained in plain language.
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New oral drug could transform life for PKU patients
Disease control Recruiting nowThis study is testing whether an oral medication called JNT-517 can safely lower harmful phenylalanine levels in adults with PKU. About 120 participants will take either the drug or a placebo twice daily for up to a year while maintaining their special diet. Researchers will meas…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. • Aim: Disease control
Last updated Apr 03, 2026 14:43 UTC
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Taste test for rare disease diets: can new protein powder make life easier?
Disease control Recruiting nowThis study is testing four new protein powder supplements designed for people with rare metabolic disorders PKU and tyrosinemia. Over 29 days, about 40 adults and children will try the powders to see if they are easier to take, taste better, and cause fewer stomach problems than …
Matched conditions: PHENYLKETONURIA
Phase: NA • Sponsor: Nutricia UK Ltd • Aim: Disease control
Last updated Mar 31, 2026 12:12 UTC
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Can a new drug protect the developing brain in kids with PKU?
Disease control Recruiting nowThis study aims to see if long-term treatment with a drug called sepiapterin can help preserve normal brain development and thinking skills in children with phenylketonuria (PKU). It will enroll 56 children, from toddlers up to age 16, who have PKU. Researchers will track changes…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated Mar 23, 2026 15:17 UTC