Gene therapy trial offers hope for kids with rare kidney disease
NCT ID NCT06910813
First seen Nov 01, 2025 · Last updated May 25, 2026 · Updated 24 times
Summary
This study tests a new gene therapy called DFT383 in children aged 2 to 5 with nephropathic cystinosis, a rare disease that damages the kidneys and other organs. The treatment aims to fix the genetic problem and reduce or eliminate the need for daily medication. About 30 children will take part, and the study will check safety and how well the therapy works over a long period.
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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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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
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
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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
Contact Phone: •••-•••-•••• Email: •••••@•••••
Contact
Conditions
Explore the condition pages connected to this study.