NCT05146830

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

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: Stephanie Cherqui · Academic / Other
• Sex: All
• Age: 14 Years – 50 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Cystinosis

Interventions

Type	Name	Description
OTHER	Safety and Efficacy Assessments	Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.

Timeline

• Start date: 2022-11-14
• Primary completion: 2036-11-30
• Completion: 2036-11-30
• First posted: 2021-12-07
• Last updated: 2024-07-03

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study