Observational study on Alport Syndrome
Study of the Natural History of Alport Syndrome by Establishment of an International Database
This study is looking at how Alport syndrome affects people over time and whether new treatments that worked in animals can help improve their health and quality of life.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 700 (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 | NCT05927467 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on Alport syndrome, a rare inherited condition that leads to kidney failure, hearing loss, and eye issues. It aims to gather comprehensive data on the progression of the disease and the effectiveness of potential therapies, including those that have shown promise in animal models. The study will utilize a European database to analyze patient outcomes and quality of life, contributing to a better understanding of the disease's natural history and informing future therapeutic trials.
Who should consider this trial
Good fit: Ideal candidates for this study are individuals diagnosed with Alport syndrome through renal biopsy or molecular studies.
Not a fit: Patients without a confirmed diagnosis of Alport syndrome will not benefit from this study.
Why it matters
Potential benefit: If successful, this study could enhance understanding of Alport syndrome and improve treatment strategies for affected patients.
How similar studies have performed: While there have been retrospective studies indicating potential treatment avenues, this study represents a novel approach to understanding the disease's progression in a European context.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosis of AS based on electron microscopic examination of the renal biopsy and/or molecular studies and/or abnormal expression of type IV collagen chains on skin and/or glomerular basement membranes. * Signed informed consent Exclusion Criteria: \- No exclusion criteria
Where this trial is running
Paris, Île-de-France Region
- RaDiCo Eurbio-Alport — Paris, Île-de-France Region, France (Recruiting)
Study contacts
- Principal investigator: Laurence Heidet, PHD — Inserm U933
- Study coordinator: Laurence Heidet, PHD
- Email: laurence.heidet@aphp.fr
- Phone: 0033 1 44 49 43 82
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.