Apply to trial NCT06910813

A few quick questions so the study team can decide if you might be a fit.

RecruitingGenetic intervention

DFT383 in Pediatric Participants With Nephropathic Cystinosis

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.

How this works

  1. Answer a few questions

    About 5 to 10 minutes. Skip-friendly where possible.

  2. We connect you with the study team

    When a site picks up your application, we introduce you to their team directly — with you on the thread.

  3. The team reaches out to schedule screening

    Usually within a few business days, via the contact you give.

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