An Open-label, Multi-center, Phase I/II Study to Assess Safety, Tolerability and Efficacy of DFT383 in Pediatric Participants With Nephropathic Cystinosis, Followed by a Long-term Extension Phase

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 I/II study also includes a long-term extension phase to gather additional data. The study includes two groups: one receiving DFT383 and another continuing standard care without the gene therapy to provide comparative data in this rare disease. Participants in the treatment group (Cohort 1) will receive DFT383 in a staggered dosing approach across three subgroups and will be involved 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 continue their standard treatment with cysteamine and participate only in the Core Phase, which lasts up to 24 months. Both cohorts will run in parallel at investigational sites. During the study, participants will undergo various assessments including measurements of kidney function, reversal of renal Fanconi syndrome, blood tests, and eye examinations for corneal cystine crystals. Researchers will monitor adverse events, quality of life, and other health indicators over time. The study aims to track these outcomes over the Core and Extension Phases, with long-term safety and efficacy being closely observed throughout the total duration of participation.

DFT383 in Pediatric Participants With Nephropathic Cystinosis