Gene therapy trial for children with cystinosis
Part of Genetic & congenital, Hormones & metabolism, Kidney & urinary, Women’s health & pregnancy clinical trials.
This trial tests a new gene therapy called DFT383 for children aged 2 to 5 with nephropathic cystinosis. It aims to see if the treatment is safe and can help improve kidney function.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 2 and 5 years old (inclusive up to 5 years and 364 days).
- You have been on oral cysteamine therapy for at least 6 months.
- You have a confirmed diagnosis of nephropathic cystinosis and signs of renal fanconi syndrome.
- Your kidney function is relatively good (eGFR of 60 or higher).
- You have received all age-appropriate vaccines.
- You have not had a kidney transplant, stem cell transplant, or gene therapy before.
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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