What this study is
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.
What's being tested
- Safety and Efficacy Assessments
Intervention · OTHER
Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.
Study details
- Sponsor
- Stephanie Cherqui
- Conditions
- Cystinosis
- Enrolling
- 50 people
- Starts
- 2022-11-14
- Est. completion
- 2036-11-30
- Record
- View on ClinicalTrials.gov ↗
Who can join
Age 14 Years – 50 YearsSex ALLHealthy volunteers Not acceptedYou may qualify if
- Participant must have received CTNS-RD-04 in a preceding study
You can't join if
- 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 Participants who have either completed, withdrawn, or prematurely discontinued participation for any reason at any time after receiving CTNS-RD-04 are eligible for CTNS RD 04 LTF01 study participation.
Sign in and we'll check these against your records.Full criteria on ClinicalTrials.gov ↗
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Study locations (1)
1 openUniversity of California San Diego
La Jolla, California, United States
Study team
Sponsor
Stephanie Cherqui
Study ID CTNS-RD-04-LTFU01
Principal investigator
Stephanie Cherqui, PhD
University of California, San Diego
Frequently asked questions
The sponsor hasn't published answers to common questions for this trial.