Classic phenylketonuria
MONDO:0019259Classical phenylketonuria is a severe form of phenylketonuria (PKU) an inborn error of amino acid metabolism characterized in untreated patients by severe intellectual deficit and neuropsychiatric complications.
Also known as: classic PKU
4 clinical trials for this condition and its sub-types, 4 tagged with Classic phenylketonuria itself.
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Can a One-Time gene infusion rewrite the fate of PKU?
Cure Recruiting nowThis early-stage trial tests a single intravenous infusion of NGGT002, a gene therapy designed to deliver a working copy of the PAH gene to adults with classic phenylketonuria (PKU). The goal is to see if this approach can safely lower phenylalanine levels in the blood, which cou…
Phase 1/2 • Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd. • Aim: Cure
Last updated Aug 15, 2026 00:00 UTC
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Can weekly Finger-Prick tests improve PKU control?
Disease control Recruiting nowResearchers are testing whether measuring phenylalanine at home once a week, rather than once a month, helps adults with classic phenylketonuria keep their levels in a healthier range. The trial enrolls 36 adults aged 18 to 65 who follow a low-protein diet and use special amino a…
Sponsor: Michał Kania • Aim: Disease control
Last updated Sep 19, 2026 00:00 UTC
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One-Time gene fix could ease PKU's strict diet burden
Disease control Recruiting nowThis early-stage trial tests a gene therapy called NGGT002 for adults with classic PKU, a genetic disorder that makes it hard to break down an amino acid called phenylalanine. The treatment delivers a working copy of the missing gene via a single IV infusion. Researchers will mon…
Phase 1/2 • Sponsor: NGGT INC. • Aim: Disease control
Last updated Aug 20, 2026 00:00 UTC