DFT383 for Cystinosis

(CYStem Trial)

This trial tests a new gene therapy called DFT383 to determine its safety and effectiveness for young children with nephropathic cystinosis, a rare condition that causes harmful cystine buildup in the body. The study consists of two parts: one group will receive DFT383, while another will continue their usual treatment for comparison. Children aged 2 to 5 years who have been on oral cysteamine therapy for at least 6 months and have nephropathic cystinosis may be suitable candidates. As a Phase 1 trial, this research aims to understand how the new gene therapy works in people, offering participants a chance to be among the first to receive this innovative treatment.

The trial does not specify if you need to stop taking your current medications, but you must have been on oral cysteamine therapy for at least 6 months. If you are taking indomethacin, you need to stop it at least 2 weeks before screening for Cohort 1.

Research has shown that DFT383 is a new type of gene therapy being tested for safety in young children with nephropathic cystinosis, a rare condition. Since testing is still in the early stages, there is limited information about its safety in humans. The study is in its first and second phases, focusing primarily on assessing safety for the children involved. These early phases typically ensure the treatment does not cause excessive side effects.

DFT383 is unique because it offers a new approach to treating cystinosis compared to the current standard of care, which typically involves cysteamine. Researchers are excited about DFT383 because it may work through a different mechanism, potentially improving how cystine is reduced in cells. This could mean better management of cystinosis symptoms and possibly fewer side effects, giving patients a new hope for improved quality of life.

Research has shown that DFT383 is a gene therapy under investigation for treating nephropathic cystinosis, a rare disease affecting kidney function. Early studies suggest that DFT383 targets the genetic issues causing the disease, potentially reducing the buildup of cystine in cells. This buildup can damage the kidneys and other organs over time. In this trial, participants will either receive DFT383 or continue with the standard of care, cysteamine. Although information on DFT383's effectiveness in people remains limited, the therapy aims to address the genetic root of the problem, potentially offering longer-lasting effects than current treatments. As more results from ongoing trials become available, they will provide clearer insights into its potential benefits.¹²⁴⁶⁷