One-Shot gene therapy aims to free PKU patients from lifelong diet
NCT ID NCT05972629
Summary
This study is testing a new, one-time gene therapy injection for adults with phenylketonuria (PKU). The goal is to see if the treatment is safe and if it can lower harmful blood Phe levels enough to allow participants to stop their highly restrictive, lifelong low-protein diet. Researchers will follow participants for at least 5 years to monitor their health and the treatment's long-term effects.
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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
Explore the condition pages connected to this study.