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
An open-label, multi-center, phase I/II study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis, followed by a long-term extension phase. The purpose of this clinical study is to assess safety, tolerability, and efficacy of DFT383 in participants aged 2 to 5 years with nephropathic cystinosis. The study consists of a Core Phase and a long-term Extension Phase. DFT383 is a cellular gene therapy. This study includes an active arm (Cohort 1) of participants treated with study treatment DFT383 and a concurrent reference arm (Cohort 0). Participants in Cohort 0 will not receive study treatment and will only participate in the Core Phase of the study. The study is not randomized and Cohort 0 aims to collect prospective and concurrent data in this rare disease.
⁃ Participants eligible for inclusion in this study must meet all the following criteria:
• Informed consent in writing from parent(s) or legal guardian(s) must be provided
• 2 to 5 years of age (including 5 years and 364 days old) at Screening
• Weight-for-stature is ≥ the third percentile, and is ≥ 10 kg
• Oral cysteamine therapy for at least 6 months
• Historic clinical diagnosis of nephropathic cystinosis
• Laboratory evidence of of renal fanconi syndrome (RFS)
• Relatively preserved kidney function (eGFR ≥ 60mL/min/1.73m2)
• Received all age-appropriate vaccinations