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 Participants who have either completed, withdrawn, or prematurely discontinued participation for any reason at any time after receiving CTNS-RD-04 are eligible for CTNS RD 04 LTF01 study participation.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) 44 research publications

Details

Status
accepting new patients by invitation only
Start Date
Completion Date
(estimated)
Sponsor
Stephanie Cherqui
ID
NCT05146830
Study Type
Observational
Participants
Expecting 50 study participants
Last Updated