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A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04
Sponsor: Stephanie Cherqui
Summary
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 Details
Gender
All
Age Range
14 Years - 50 Years
Study Type
OBSERVATIONAL
Enrollment
50
Start Date
2022-11-14
Completion Date
2036-11-30
Last Updated
2024-07-03
Healthy Volunteers
No
Conditions
Interventions
Safety and Efficacy Assessments
Safety evaluations, disease-specific assessments, and other assessments to monitor for long-term complications of gene therapy intervention.
Locations (1)
University of California San Diego
La Jolla, California, United States