Gene therapy may free kids from daily cystinosis meds
NCT ID NCT06910813
First seen Jun 27, 2026 · Last updated Jul 31, 2026 · Updated 3 times
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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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
Locations
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Baylor College of Medicine - Texas Children's Hospital (recuiting Cohort 0)
RECRUITINGHouston, Texas, 77030, United States
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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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Phoenix Children's Hospital (Recruitng Cohort 0 and 1)
NOT_YET_RECRUITINGPhoenix, Arizona, 85016, United States
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Stanford University - Stanford Children's Health
RECRUITINGStanford, California, 94305, United States
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University of California at San Diego - Rady Children's Hospital
RECRUITINGSan Diego, California, 92123, United States
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Other studies related to the condition(s) this trial covers.