Cystinosis clinical trials at University of California Health

2 in progress, 1 open to eligible people

Showing trials for

  • DFT383 in Pediatric Participants With Nephropathic Cystinosis

    open to eligible people ages 2-5

    An open-label, multi-center, phase I/II study to assess the safety, tolerability and efficacy of DFT383 in pediatric participants with nephropathic cystinosis. 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. 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) treated with Standard of care (SoC). The study is not randomized and Cohort 0 aims to collect prospective and concurrent data in this rare disease.

    at UCSD

  • Participants With Cystinosis Who Previously Received CTNS-RD-04

    Sorry, accepting new patients by invitation only

    This is a multinational, long-term follow-up study to assess the long-term safety and durability of CTNS-RD-04 treatment in participants who received a single dose administration of lentiviral gene therapy. No investigational product will be administered in this study. Participants will continue periodic safety and efficacy assessments in this long-term follow-up study up to 15 years from the initial date of CTNS-RD-04 infusion.

    at UCSD

Our lead scientists for Cystinosis research studies include .

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