PHENYLKETONURIA (PKU)
Clinical trials for PHENYLKETONURIA (PKU) explained in plain language.
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Experimental enzyme injection aims to tame PKU's toxic amino acid
Disease control Recruiting nowThis early-stage trial tests an experimental drug called PL54 in adults aged 18 to 55 with phenylketonuria (PKU), a genetic disorder that prevents the body from breaking down the amino acid phenylalanine. PL54 is an enzyme designed to help lower high blood phenylalanine levels, w…
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE1 • Sponsor: Chongqing Peg-Bio Biopharm Co., Ltd. • Aim: Disease control
Last updated Jul 02, 2026 04:00 UTC
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New PKU drug JNT-517 moves to Long-Term safety trial
Disease control Recruiting nowThis Phase 3 study is testing the long-term safety of an oral drug called JNT-517 in 240 children and adults with phenylketonuria (PKU). All participants will receive the drug twice daily, with doses based on age and weight. The goal is to see if JNT-517 is safe to use over time …
Matched conditions: PHENYLKETONURIA (PKU)
Phase: PHASE3 • Sponsor: Otsuka Pharmaceutical Development & Commercialization, Inc. • Aim: Disease control
Last updated Jun 27, 2026 12:28 UTC
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10-Year study to track immune risks of PKU drug
Disease control Recruiting nowThis 10-year study will follow 200 people with PKU who are taking or about to start Pegvaliase (Palynziq). Researchers will monitor immune reactions, inflammation, and lab results to better understand long-term safety. The goal is to improve management of side effects like allerg…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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10-Year study tracks safety of PKU drug palynziq
Disease control Recruiting nowThis 10-year observational study is following 450 people with PKU who are taking or starting pegvaliase (Palynziq) to monitor long-term safety, especially allergic reactions. Researchers are tracking serious side effects like anaphylaxis, severe joint pain, and low phenylalanine …
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: BioMarin Pharmaceutical • Aim: Disease control
Last updated Jun 27, 2026 12:00 UTC
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PKU gene study seeks clues in adult health
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 samples from 149 participants to find patterns that may explain differences in health and thinking skills. No…
Matched conditions: PHENYLKETONURIA (PKU)
Sponsor: University Hospital, Tours • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:28 UTC