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

Plain-language summary

This study provides long-term follow-up of patients with cystinosis who previously received the CTNS-RD-04 gene therapy, and evaluates the safety and effect of the treatment. It includes participants who were given CTNS-RD-04 in a preceding study. This study has no assigned phase and is enrolling by invitation.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Enrolling by invitation

Sponsor

Stephanie Cherqui

Intervention

Safety and Efficacy Assessments (Gene Therapy Intervention)

Start date

11/14/2022

Estimated completion

11/30/2036

Countries

United States

Related genes

Locations

• La Jolla, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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 Participants who have either completed, withdrawn, or prematurely discontinued participation for any reason at any time after receiving CTNS-RD-04 are eligible for CTNS RD 04 LTF01 study participation.