PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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New PKU drug JNT-517 moves to Long-Term safety check in phase 3 trial
Disease control Recruiting nowThis study tests the long-term safety of an experimental drug called JNT-517 in children and adults with PKU, a genetic condition that causes harmful buildup of phenylalanine. All 240 participants will receive the drug, and researchers will monitor side effects and changes in blo…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE3 • Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. • Aim: Disease control
Last updated May 15, 2026 11:55 UTC
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10-Year study tracks safety of PKU drug palynziq
Disease control Recruiting nowThis study follows 450 people with phenylketonuria (PKU) who are taking or starting the drug Palynziq. Over 10 years, researchers will monitor for allergic reactions and other side effects to better understand the drug's long-term safety. Participants receive their usual treatmen…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 11, 2026 20:48 UTC
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PKU puzzle: scientists hunt for Gene-Metabolism links in adults
Knowledge-focused Recruiting nowThis study looks at how genes and body chemistry are connected in 149 adults who were treated for phenylketonuria (PKU) from birth. Researchers will analyze blood and saliva samples to find patterns that might explain differences in health and thinking skills. The goal is to bett…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: University Hospital, Tours • Aim: Knowledge-focused
Last updated May 12, 2026 13:43 UTC
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10-Year PKU drug safety study launches to track immune reactions
Knowledge-focused Recruiting nowThis study follows 200 people with PKU who are taking or about to start Palynziq (pegvaliase) for up to 10 years. Researchers will track immune responses, inflammation, and lab results to better understand long-term side effects like allergic reactions and joint pain. The goal is…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: BioMarin Pharmaceutical • Aim: Knowledge-focused
Last updated May 04, 2026 16:22 UTC