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European Cystinosis Cohort 2

NCT07680751 · Status: NOT_YET_RECRUITING · Type: OBSERVATIONAL · Enrollment: 250

Last updated 2026-07-02

No results posted yet for this study

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.

Conditions

  • Cystinosis

Sponsors & Collaborators

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

    lead OTHER_GOV

Principal Investigators

  • Aude Servais · AP-HP_Hôpital Necker_Paris

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-07-01
Primary Completion
2028-03-01
Completion
2028-03-01

Countries

  • France

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07680751 on ClinicalTrials.gov