National registry for patients with bone marrow failure syndromes in France
French National Registry of Bone Marrow Failures: Prospective and Retrospective Database Associated to a Collection of Biological Samples: RIME Project
This study is collecting health information and samples from patients with bone marrow failure syndromes in France to better understand these rare diseases and improve their treatment.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 5000 (estimated) |
| Sex | All |
| Sponsor | Assistance Publique - Hôpitaux de Paris Academic / other |
| Locations | 1 site (Paris) |
| Trial ID | NCT04781790 on ClinicalTrials.gov |
What this trial studies
This observational study collects standardized clinical information from patients diagnosed with bone marrow failure syndromes across multiple centers in France. It aims to gather data from diagnosis through follow-up, regardless of treatment status, to better understand the natural history of these rare diseases. Additionally, biological samples such as blood, bone marrow, and skin are collected and biobanked for future translational research. The registry seeks to evaluate public health needs and improve the management of these conditions based on real-life efficacy and guidelines.
Who should consider this trial
Good fit: Ideal candidates include individuals of all ages diagnosed with any form of bone marrow failure syndrome who consent to participate.
Not a fit: Patients with myelodysplastic syndrome over the age of 50 without genetic predispositions or familial forms may not benefit from this study.
Why it matters
Potential benefit: If successful, this registry could enhance the understanding and management of bone marrow failure syndromes, leading to improved patient outcomes.
How similar studies have performed: While this registry approach is not novel, it builds on existing frameworks for collecting clinical data and biological samples, which have shown success in other rare disease contexts.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * All age * All diagnostic of BMF * Having given his non-opposition to registry after understand overall aims * Having signed a written informed consent (2 parents for patients aged less than 18) for collection of biological samples * With health insurance coverage Exclusion Criteria: With myelodysplastic syndrome occurring in a patient over the age of 50 in absence of genetical predispositions, familial forms and history of medullary hypoplasia
Where this trial is running
Paris
- Hématologie Greffe — Paris, France (Recruiting)
Study contacts
- Study coordinator: Régis Peffault De Latour
- Email: regis.peffaultdelatour@aphp.fr
- Phone: +33142385073
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.