Actively Recruiting

All Genders
NCT04781790

French National Registry of Bone Marrow Failures

Led by Assistance Publique - Hôpitaux de Paris · Updated on 2021-03-04

5000

Participants Needed

1

Research Sites

521 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This is a unique clinical and biological database that collects standardized clinical information during the management of all patients with bone marrow failure syndromes (BMF) in France (multicenter registry), from diagnosis and throughout follow-up during the natural history of the disease, treated or not. In parallel, biological samples (blood and/or bone marrow and/or skin) are collected during clinical care and are biobanked in Saint-Louis Hospital (Hematology laboratory) in order to be used in translational research related to bone marrow failure diseases. This registry has two main objectives: * Public health care evaluation and improvement: to assess the medical and social needs inherent to the management of these rare diseases; to precisely assess the level of diagnosis and management of bone marrow failure syndromes in France; to evaluate the impact and guidance of the French reference center guidelines for diagnosis and treatment; to evaluate the real-life efficacy and tolerance of any given specific treatments; to analyze treatment's cost-effectiveness according to each situation. * Research: * Epidemiology: to determine the incidence, prevalence, and distribution of different bone marrow failure syndromes at the national level; * Biology: to better understand the pathophysiology of BMF; to identify and to study complications within each entity, such as mechanisms underlying clonal evolution, new forms of inherited BMF and acute myeloid leukemia (AML)/MDS-predisposition syndromes, and to better and deeper characterize known entities; * Treatment: to identify prognostic factors and predictors of response; to identify side effects and impact of treatment on others organs and natural functions; to assess patients' quality of life as early as possible since diagnosis and throughout follow-up.

CONDITIONS

Official Title

French National Registry of Bone Marrow Failures

Who Can Participate

All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • All ages
  • Diagnosis of bone marrow failure syndrome
  • Non-opposition to registry participation after understanding study aims
  • Signed written informed consent (both parents for patients under 18) for biological sample collection
  • Health insurance coverage
Not Eligible

You will not qualify if you...

  • Myelodysplastic syndrome in patients over 50 without genetic predispositions, familial forms, or history of medullary hypoplasia

AI-Screening

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Trial Site Locations

Total: 1 location

1

Hématologie Greffe

Paris, France

Actively Recruiting

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Research Team

R

Régis Peffault De Latour

CONTACT

M

Matthieu Resche-Rigon

CONTACT

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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