Gene therapy may free kids from daily cystinosis meds
NCT ID NCT06910813
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a gene therapy called DFT383 in 30 children aged 2 to 5 with nephropathic cystinosis, a rare disease that damages kidneys and other organs. The treatment aims to fix the genetic problem so children may no longer need daily cysteamine medication. Researchers will check safety and whether the therapy can reverse kidney problems.
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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
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Contact
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