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

Part of paid clinical trials in La Jolla, California.

Sponsor
Stephanie Cherqui
Study ID
NCT05146830
Status
Enrolling By Invitation

Conditions

  • Cystinosis

Eligibility Criteria

Sex
ALL
Age
14 Years - 50 Years
Healthy Volunteers
Not accepted

Interventions

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

Study Details

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.

Key Dates

Start date
Nov 14, 2022
Status verified
Jul 2024
Primary completion
Nov 30, 2036
Completion
Nov 30, 2036

Study Design

Enrollment
50 participants (estimated)

Arms

  • Arm: Participants with Cystinosis Disease
    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.

Primary Outcome Measure

Incidence of clinically significant Adverse Events (AEs) and Serious Adverse Events (SAEs) [ Time Frame: Baseline to Year 15 post gene therapy ]

Locations (1)

FacilityCityStateZIPSite coordinators
University of California San DiegoLa JollaCalifornia92037-

Find similar trials in La Jolla, CA

By research site
University of California San Diego· La Jolla, CA

Related Studies