National registry for patients with bone marrow failure syndromes in France

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Hématologie Greffe
  Paris, France Recruiting

Study contacts

• Study coordinator: Régis Peffault De Latour
• Email: regis.peffaultdelatour@aphp.fr
• Phone: +33142385073

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.