Cohort study on cystinosis in Europe
European Cystinosis Cohort
This study looks at how cystinosis affects the lives of patients in Europe and how different treatments impact their health and well-being.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 400 (estimated) |
| Sex | All |
| Sponsor | Institut National de la Santé Et de la Recherche Médicale, France Government |
| Locations | 1 site (Paris, Île-de-France Region) |
| Trial ID | NCT05901077 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on cystinosis, a rare lysosomal storage disease affecting multiple organs due to cystine accumulation. It aims to assess the quality of life and the effects of treatments in patients diagnosed with cystinosis across Europe. The study will involve patients with confirmed diagnoses, who will provide informed consent to participate. By gathering data on the experiences and health outcomes of these patients, the study seeks to enhance understanding of the disease and improve care strategies.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals with a confirmed diagnosis of cystinosis who can provide informed consent.
Not a fit: Patients who are unable to give informed consent or those with associated diseases may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to improved management and treatment strategies for patients with cystinosis, enhancing their quality of life.
How similar studies have performed: While there have been studies on cystinosis, this cohort approach focusing on quality of life and treatment effects is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Confirmed diagnosis of cystinosis (based on cystine dosage, presence of crystals at eye examination or molecular diagnosis) * Signed informed consent Exclusion Criteria: * Patients not able to give their informed consent. No other criteria (patients with associated disease should be enrolled).
Where this trial is running
Paris, Île-de-France Region
- RaDiCo-ECYSCO — Paris, Île-de-France Region, France (Recruiting)
Study contacts
- Principal investigator: Aude Servais, PHD — Inserm U933
- Study coordinator: Aude Servais, PHD
- Email: aude.servais@aphp.fr
- Phone: 0033 1 44 38 15 15
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.