Summary

Eligibility
for people ages 14-50 (full criteria)
Location
at La Jolla, California
Dates
study started
completion around
Principal Investigator
by Stephanie Cherqui, PhD

Description

Summary

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.

Details

Participants enrolled in a study where the individual received CTNS-RD-04 will be offered participation in the CTNS-RD-04-LTF01 study. The Baseline visit for the CTNS-RD-04-LTF01 study will likely coincide with the final visit in the parent study. Participants confirmed eligible for the CTNS-RD-04-LTF01 study will be asked to return for study visits at approximately 6-month intervals for the first 4 years and annually thereafter for up to 11 years until a total of 15 years have elapsed during which time continued safety, engraftment, and efficacy of CTNS-RD-04 treatment will be assessed.

Keywords

Cystinosis, Cystinosis Disease, Cell therapy, Gene therapy, Long-term follow-up, Safety and Efficacy Assessments

Eligibility

You can join if…

Open to people ages 14-50

• Participant must have received CTNS-RD-04 in a preceding study

You CAN'T join if...

• Participant is currently enrolled in an CTNS-RD-04 treatment study. Participants who have either completed, withdrawn, or prematurely discontinued participation for any reason at any time after receiving CTNS-RD-04 are eligible for CTNS RD 04 LTF01 study participation.

Location

  • University of California San Diego
    La Jolla California 92037 United States

Lead Scientist at UCSD

  • Stephanie Cherqui, PhD
    Professor, Pediatrics, Vc-health Sciences-schools. Authored (or co-authored) 43 research publications

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
University of California, San Diego
ID
NCT05146830
Study Type
Observational
Participants
Expecting 50 study participants
Last Updated