Phase III Study of DFT383 Gene Therapy for Children Ages 2 to 5 with Nephropathic Cystinosis Including Long-term Follow-up

What this Trial Is About

Researchers are evaluating the safety, tolerability, and effectiveness of DFT383, a cellular gene therapy, in children aged 2 to 5 years with nephropathic cystinosis. This open-label, multi-center, phase III study includes a Core Phase and a long-term Extension Phase to gather detailed data on this rare disease. The study features two groups one receiving DFT383 and another continuing with standard care for comparison. Participants in the DFT383 group Cohort 1 will receive the gene therapy in a staggered dosing approach across three sub-cohorts. This group will participate in both the Core Phase lasting up to 32 months and a long-term Extension Phase lasting up to 13 years. The comparison group Cohort 0 will not receive the gene therapy but will continue their usual cysteamine treatment during the Core Phase, which lasts up to 24 months. Throughout the study, participants will undergo regular monitoring including safety assessments, kidney function tests, and evaluations of renal Fanconi syndrome reversal. Researchers will also track quality of life and long-term health outcomes such as kidney failure and malignancy. The study includes frequent laboratory tests, vital signs checks, and corneal crystal assessments, with extended follow-up to understand long-term effects of DFT383 and standard care treatments.

DFT383 in Pediatric Participants With Nephropathic Cystinosis