Cystinosis clinical trials at University of California Health

2 in progress, 0 open to eligible people

Showing trials for

  • Stem Cell Gene Therapy for Cystinosis

    Sorry, in progress, not accepting new patients

    This study is a Phase 1/2 clinical trial that will assess the safety and efficacy of enriched gene-corrected hematopoietic stem cells isolated from patients affected with cystinosis. (Investigational Product: CTNS-RD-04 or CTNS-RD-04-LB, where the suffix "-LB" stands for LentiBOOST)

    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 .