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 for children and adults with phenylketonuria (PKU), a genetic condition that causes harmful buildup of phenylalanine. About 240 participants who completed earlier JNT-517 studies or are new to the drug w…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE3 • Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. • Aim: Disease control
Last updated May 22, 2026 13:54 UTC
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10-Year study to monitor safety of PKU drug palynziq
Disease control Recruiting nowThis study follows 450 people with phenylketonuria (PKU) who are taking or starting Palynziq injections. Over 10 years, researchers will track side effects like allergic reactions and joint pain to better understand the drug's long-term safety. The goal is to improve real-world u…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated May 20, 2026 11:55 UTC
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10-Year study tracks immune reactions in PKU patients on pegvaliase
Knowledge-focused Recruiting nowThis study follows 200 people with PKU who are already taking or about to start pegvaliase. Over 10 years, researchers will monitor immune responses and lab results to better understand long-term safety. The goal is to learn, not to test a new treatment.
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: BioMarin Pharmaceutical • Aim: Knowledge-focused
Last updated May 26, 2026 08:33 UTC
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PKU gene hunt: can DNA explain brain health in adults?
Knowledge-focused Recruiting nowThis study looks at how genes and body chemistry are linked in adults with phenylketonuria (PKU) who were treated early in life. Researchers will analyze blood and saliva from 149 participants to find patterns in metabolism and DNA. The goal is to better understand the disease, n…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: University Hospital, Tours • Aim: Knowledge-focused
Last updated May 20, 2026 11:54 UTC