PHENYLKETONURIA
Clinical trials for PHENYLKETONURIA explained in plain language.
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New protein powders could make PKU diet easier and tastier
Disease control Recruiting nowThis study is testing four new powdered protein substitutes for people with PKU or tyrosinemia, rare conditions that require a special diet. The products are made from a milk protein called GMP and are designed to be more palatable and easier to stick with than current options. A…
Matched conditions: PHENYLKETONURIA
Phase: NA • Sponsor: Nutricia UK Ltd • Aim: Disease control
Last updated May 26, 2026 04:53 UTC
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New PKU drug enters early human 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 the amino acid phenylalanine. The main goal is to check the drug's safety and how the body processes it. Participants must have a specific…
Matched conditions: PHENYLKETONURIA
Phase: PHASE1 • Sponsor: Agios Pharmaceuticals, Inc. • Aim: Disease control
Last updated May 22, 2026 13:54 UTC
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Can a daily pill preserve IQ in kids with PKU? new trial aims to find out
Disease control Recruiting nowThis study tests whether the drug sepiapterin can help preserve thinking and learning skills in children with phenylketonuria (PKU) when started in early childhood. About 56 children will take the medicine and have their IQ and quality of life measured over time. The goal is to s…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 22, 2026 13:52 UTC
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New pill could help control PKU without strict diet
Disease control Recruiting nowThis study tests an oral medication called JNT-517 for adults with phenylketonuria (PKU), a genetic condition that makes it hard to break down an amino acid called phenylalanine. About 120 adults will take either JNT-517 or a placebo for 6 weeks, then everyone gets JNT-517 for al…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. • Aim: Disease control
Last updated May 19, 2026 11:59 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 affect amino acid levels and metabolic control 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-exer…
Matched conditions: PHENYLKETONURIA
Phase: NA • Sponsor: Hacettepe University • Aim: Knowledge-focused
Last updated May 26, 2026 04:35 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 18, 2026 12:08 UTC