One-Time gene therapy could free PKU patients from strict diet
NCT ID NCT05972629
First seen Nov 01, 2025 · Last updated May 16, 2026 · Updated 27 times
Summary
This study tests a one-time gene therapy (SAR444836) for adults with phenylketonuria (PKU), a genetic disorder that makes it hard to break down an amino acid called phenylalanine (Phe). The goal is to lower Phe levels in the blood and allow participants to eat a normal diet without strict restrictions. About 32 adults will receive the treatment and be followed for at least 5 years to check safety and effectiveness.
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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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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
Conditions
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