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

Stephanie Cherqui

Status: Invitation-only

Conditions: Cystinosis

Treatments: Other: Safety and Efficacy Assessments

Study type: Observational

Funder types: Other; Industry

Identifiers

NCT05146830

CTNS-RD-04-LTFU01

Details and patient eligibility

About

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.

Full 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.

Enrollment: 50 estimated patients

Sex: All

Ages: 14 to 50 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

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

Exclusion criteria

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 Participants who have either completed, withdrawn, or prematurely discontinued participation for any reason at any time after receiving CTNS-RD-04 are eligible for CTNS RD 04 LTF01 study participation.

Trial design

50 participants in 1 patient group

Participants with Cystinosis Disease

Description:

This is a long-term follow-up study of participants who previously received CTNS-RD-04 (single dose administration). No investigational product will be administered in this study.

Treatment:

Other: Safety and Efficacy Assessments