French National Registry of Bone Marrow Failures

NCT ID: NCT04781790

Last Updated: 2021-03-04

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 5000 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2017-02-06
• Study Completion Date: 2027-02-06

Brief Summary

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

Bone Marrow Failure Syndrome

Study Design

• Observational Model Type: OTHER
• Study Time Perspective: PROSPECTIVE

Study Groups

Patients with Bone marrow failure

Standard of care of patients with bone marrow failure

Bone Marrow Failure

Intervention Type: OTHER

without interventional intervention . biological collection in the routine care Standard of care of patients with bone marrow failure.

Interventions

Bone Marrow Failure

without interventional intervention . biological collection in the routine care Standard of care of patients with bone marrow failure.

Intervention Type: OTHER

Eligibility 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

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Hématologie Greffe

Paris, , France

Site Status: RECRUITING

Countries

France

Central Contacts

Régis Peffault De Latour

Role: CONTACT

regis.peffaultdelatour@aphp.fr

+33142385073

Matthieu Resche-Rigon

Role: CONTACT

matthieu.resche-rigon@u-paris.fr

+3341499742

Facility Contacts

Regis PEFFAULT DE LATOUR

Role: primary

Other Identifiers

NI17045J

Identifier Type: -

Identifier Source: org_study_id