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

First listed
Dec 7, 2021
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

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