PHENYLKETONURIA
Clinical trials for PHENYLKETONURIA explained in plain language.
Never miss a new study
Get alerted when new PHENYLKETONURIA trials appear
Sign up with your email to follow new studies for PHENYLKETONURIA, keep track of the ones that matter, and come back to a personal dashboard instead of checking manually.
Genom att skicka in godkänner du våra Användarvillkor
-
New PKU drug shows promise for easier diet management in Long-Term trial
Disease control OngoingThis study tests the long-term safety of an experimental drug called PTC923 (sepiapterin) in about 200 people with phenylketonuria (PKU), a genetic condition that makes it hard to process the amino acid phenylalanine. The main goals are to check for side effects and see if the dr…
Matched conditions: PHENYLKETONURIA
Phase: PHASE3 • Sponsor: PTC Therapeutics • Aim: Disease control
Last updated May 26, 2026 11:31 UTC
-
One-Time gene therapy could free PKU patients from strict diet
Disease control OngoingThis study tests a one-time gene therapy (SAR444836) for adults with phenylketonuria (PKU), a genetic disorder causing high phenylalanine levels that require a strict diet. The treatment aims to safely lower these levels and allow patients to eat more freely. About 32 adults will…
Matched conditions: PHENYLKETONURIA
Phase: PHASE1, PHASE2 • Sponsor: Sanofi • Aim: Disease control
Last updated May 26, 2026 11:08 UTC
-
Quick fix for drug allergies: new study aims to keep PKU patients on treatment
Symptom relief OngoingThis study tests a one-day rapid drug desensitization (RDD) procedure in 9 adults with phenylketonuria (PKU) who have had allergic reactions to their medication Palynziq. The goal is to see if RDD allows them to restart and stay on Palynziq without severe allergic reactions, help…
Matched conditions: PHENYLKETONURIA
Phase: PHASE4 • Sponsor: BioMarin Pharmaceutical • Aim: Symptom relief
Last updated May 26, 2026 11:30 UTC