One-Time gene therapy aims to cure devastating childhood kidney disease
NCT ID NCT06910813
Summary
This study is testing a one-time gene therapy called DFT383 in young children (ages 2-5) with nephropathic cystinosis, a rare and serious genetic disease that damages the kidneys and other organs. The main goals are to see if the treatment is safe and if it can reverse kidney damage and free children from needing daily medication. About 15 children will receive the gene therapy, while another 15 will be observed for comparison.
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Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Locations
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Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0)
RECRUITINGHouston, Texas, 77030, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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Emory University School of Medicine - Children's Healthcare of Atlanta (recuiting Cohort 0)
RECRUITINGAtlanta, Georgia, 30322, United States
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Stanford University - Stanford Children's Health
RECRUITINGStanford, California, 94305, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California at San Diego - Rady Children's Hospital
RECRUITINGSan Diego, California, 92123, United States
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Conditions
Explore the condition pages connected to this study.