One-Time gene therapy could free PKU patients from strict diet

NCT ID NCT05972629

Ongoing Disease control Sponsor: Sanofi Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 26, 2026 · Updated 2 times

Summary

This study tests a one-time gene therapy called SAR444836 for adults with phenylketonuria (PKU), a genetic disorder that makes it hard to break down an amino acid called phenylalanine. The therapy uses a harmless virus to deliver a working copy of the missing gene. Researchers want to see if it safely lowers phenylalanine levels in the blood and allows participants to eat a normal diet without strict protein restrictions. The trial involves 32 adults and will follow them for at least 5 years.

What this could mean

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

Active substance: SAR444836 (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 allow people with PKU to eat a normal diet without strict protein restrictions, by helping their bodies break down phenylalanine.
What could go wrong: This is an early phase 1/2 trial with only 32 participants, so safety and effectiveness are not yet proven. Gene therapies can cause immune reactions or may not work for everyone, and long-term effects are unknown.

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.

phenylketonuria

As listed by the trial registrant

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

PHENYLKETONURIA

Contacts and locations

Locations

Children's Hospital IMD Clinic- Site Number : 8400015

Aurora, Colorado, 80045, United States
Hospital de Clinicas de Porto Alegre - HCPA- Site Number : 0760001

Porto Alegre, Rio Grande do Sul, 90035903, Brazil
Investigational Site Number : 0320002

Ciudad Autonoma Buenos Aires, Buenos Aires, 1199, Argentina
Investigational Site Number : 3760001

Tel Litwinsky, 52621, Israel
Investigational Site Number : 7920001

Ankara, 06560, Turkey (Türkiye)
Medical University of SC- Site Number : 8400004

Charleston, South Carolina, 29425, United States
UHCMC- Site Number : 8400014

Cleveland, Ohio, 44106, United States
UPMC Children's Hospital of Pittsburgh-4401 Penn Ave- Site Number : 8400018

Pittsburgh, Pennsylvania, 15224-1334, United States
University of California San Francisco- Site Number : 8400007

San Francisco, California, 94143, United States
University of Florida-Genetics- Site Number : 8400010

Gainesville, Florida, 32610, United States
University of Texas- Site Number : 8400002

Houston, Texas, 77030, United States

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