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A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

Cystinosis Clinical Trial

Official title:
A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

Clinical Trial 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.

Clinical Trial Description

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. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT05146830
Study type Observational
Source University of California, San Diego
Contact
Status Enrolling by invitation
Phase
Start date November 14, 2022
Completion date November 30, 2036
View Details
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