PHENYLKETONURIA
Clinical trials for PHENYLKETONURIA explained in plain language.
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Can a daily pill save kids' brains from PKU damage?
Disease control Recruiting nowThis study tests a drug called sepiapterin in 56 children with phenylketonuria (PKU), a genetic condition that can harm the brain if not managed. The goal is to see if starting treatment early can preserve thinking skills like IQ and quality of life over the long term. Children a…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 17, 2026 08:47 UTC
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New pill could help control PKU without strict diet
Disease control Recruiting nowThis study tests an oral drug called JNT-517 for adults with phenylketonuria (PKU), a genetic condition that causes harmful buildup of phenylalanine (Phe). About 120 adults will take either JNT-517 or a placebo for 6 weeks, then all will receive JNT-517 for nearly a year. The goa…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. • Aim: Disease control
Last updated May 15, 2026 11:55 UTC
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New protein powders could make life easier for people with rare metabolic disorders
Disease control Recruiting nowThis study is testing four new powdered protein substitutes for people with phenylketonuria (PKU) or tyrosinemia (TYR). These products are designed to be more palatable and easier to tolerate than current options, potentially improving diet compliance. The study will enroll 40 ad…
Matched conditions: PHENYLKETONURIA
Phase: NA • Sponsor: Nutricia UK Ltd • Aim: Disease control
Last updated May 14, 2026 12:06 UTC
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New PKU drug AG-181 enters early human safety testing
Disease control Recruiting nowThis early-stage study tests a new drug called AG-181 in 20 adults with phenylketonuria (PKU), a genetic condition that causes high levels of phenylalanine in the blood. The main goal is to check if the drug is safe and tolerable. Researchers will also measure how the drug moves …
Matched conditions: PHENYLKETONURIA
Phase: PHASE1 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 11, 2026 20:51 UTC
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Metabolic camp aims to boost pregnancy health in rare genetic disorders
Knowledge-focused Recruiting nowThis study looks at whether a camp that teaches teens and young women with PKU or MSUD about their condition and nutrition can improve their pregnancy outcomes and quality of life. Researchers will compare the results of women who attended the camp to those who did not. The study…
Matched conditions: PHENYLKETONURIA
Phase: NA • Sponsor: Emory University • Aim: Knowledge-focused
Last updated May 17, 2026 08:47 UTC
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Pre-Exercise meals studied for PKU patients
Knowledge-focused Recruiting nowThis study looks at how different types of meals before exercise change amino acid levels in adults with phenylketonuria (PKU). Ten participants will do aerobic and resistance exercises after eating different test meals. The goal is to find the best pre-exercise meal to help main…
Matched conditions: PHENYLKETONURIA
Phase: NA • Sponsor: Hacettepe University • Aim: Knowledge-focused
Last updated May 15, 2026 11:55 UTC