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European Cystinosis Cohort 2
Sponsor: Institut National de la Santé Et de la Recherche Médicale, France
Summary
This European observational cohort follows patients with cystinosis, a rare lysosomal storage disease caused by CTNS mutations leading to cystine accumulation and multisystem involvement. It aims to describe the long-term clinical course under current treatments, focusing on renal and extra-renal complications, survival, and quality of life. It also evaluates treatment effects and explores biomarkers, including inflammatory markers, with biobanking for future research.
Key Details
Gender
All
Age Range
Any - Any
Study Type
OBSERVATIONAL
Enrollment
250
Start Date
2026-07-01
Completion Date
2028-03-01
Last Updated
2026-07-02
Healthy Volunteers
No
Conditions
Locations (1)
AP-HP_ Hôpital Charles Foix
Ivry-sur-Seine, Île-de-France Region, France