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
3 clinical trials for this condition and its sub-types.
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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: PHASE1, PHASE2 • Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd. • Aim: ⭐️ CURE ⭐️
Last updated Aug 15, 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: PHASE1, PHASE2 • Sponsor: NGGT INC. • Aim: Disease control
Last updated Jun 27, 2026 08:01 UTC
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What should PKU patients eat before exercise? new study aims to find out
Knowledge-focused Recruiting nowThis study looks at how different meals before exercise affect amino acid levels in adults with phenylketonuria (PKU). Ten participants will try water, carbohydrates, or protein before doing aerobic and resistance exercises. Researchers will measure changes in blood amino acids t…
Phase: NA • Sponsor: Hacettepe University • Aim: Knowledge-focused
Last updated Jun 27, 2026 12:08 UTC