European Cystinosis Cohort 2

Starting soon

Conditions studied: Cystinosis

In brief

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 facts

Study ID

NCT07680751

Run by

Institut National de la Santé Et de la Recherche Médicale, France

People needed

250

Starts

2026-07-01

Expected to finish

2028-03-01

Last updated by the study team

2026-07-02

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• Confirmed diagnosis of cystinosis based on leukocyte cystine measurement, presence of corneal cystine crystals, and/or molecular genetic diagnosis
• Signed informed consent obtained from the patient or legal representative

You may not qualify if…

• Patients unable to provide informed consent or without a legal representative when required
• No other specific exclusion criteria; patients with associated diseases may be included

Where it is running

• AP-HP_ Hôpital Charles Foix — Ivry-sur-Seine, Île-de-France Region, France

Full record on ClinicalTrials.gov

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