One-Time gene therapy could free PKU patients from strict diet
NCT ID NCT05972629
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.
Disclaimer
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the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Children's Hospital IMD Clinic- Site Number : 8400015
Aurora, Colorado, 80045, United States
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Hospital de Clinicas de Porto Alegre - HCPA- Site Number : 0760001
Porto Alegre, Rio Grande do Sul, 90035903, Brazil
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Investigational Site Number : 0320002
Ciudad Autonoma Buenos Aires, Buenos Aires, 1199, Argentina
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Investigational Site Number : 3760001
Tel Litwinsky, 52621, Israel
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Investigational Site Number : 7920001
Ankara, 06560, Turkey (Türkiye)
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Medical University of SC- Site Number : 8400004
Charleston, South Carolina, 29425, United States
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UHCMC- Site Number : 8400014
Cleveland, Ohio, 44106, United States
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UPMC Children's Hospital of Pittsburgh-4401 Penn Ave- Site Number : 8400018
Pittsburgh, Pennsylvania, 15224-1334, United States
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University of California San Francisco- Site Number : 8400007
San Francisco, California, 94143, United States
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University of Florida-Genetics- Site Number : 8400010
Gainesville, Florida, 32610, United States
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University of Texas- Site Number : 8400002
Houston, Texas, 77030, United States