Cystinosis clinical trials at UCSD
2 in progress, 0 open to eligible people
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)
La Jolla, California
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.
La Jolla, California
Our lead scientists for Cystinosis research studies include Stephanie Cherqui, Ph.D..