Can a One-Time gene infusion rewrite the fate of PKU?

NCT ID NCT06687733

Recruiting now ⭐️ CURE ⭐️ Sponsor: NGGT (Suzhou) Biotechnology Co., Ltd. Source: ClinicalTrials.gov ↗

First seen Aug 13, 2026 · Last updated Aug 14, 2026 · Updated 1 time

Summary

This 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 could reduce the need for a strict low-protein diet. Participants will be monitored for five years to assess long-term safety and effectiveness.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: NGGT002, a gene therapy using a harmless virus to deliver a working copy of the PAH gene
What this could lead to: If successful, this one-time gene therapy could provide a lasting treatment for PKU, potentially freeing patients from strict dietary restrictions.
What could go wrong: This is an early-stage trial with only 18 participants, so safety and effectiveness are not yet proven. Gene therapies carry risks like immune reactions or the therapy not working as intended.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

classic phenylketonuria phenylketonuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PHENYLKETONURIAS

Contacts and locations

Locations

First Affiliated Hospital of Bengbu Medical College
RECRUITING

Bengbu, Anhui, China
Xinhua Hospital Affifiated to Shanghai Jiao Tong University School of Medicine
RECRUITING

Shanghai, China

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