Ignite Creation Date:
2024-05-06 @ 3:51 PM
Last Modification Date:
2024-10-26 @ 1:58 PM
Study NCT ID:
NCT04781790
Status:
RECRUITING
Last Update Posted:
2021-03-04
First Post:
2021-03-01
Brief Title:
French National Registry of Bone Marrow Failures
Sponsor:
Assistance Publique - Hôpitaux de Paris
Organization:
Assistance Publique - Hôpitaux de Paris
Study Overview
Official Title:
French National Registry of Bone Marrow Failures Prospective and Retrospective Database Associated to a Collection of Biological Samples RIME Project
Status:
RECRUITING
Status Verified Date:
2021-02
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
RIME
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 andor bone marrow andor 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 treatments 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 AMLMDS-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
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 treatments 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 AMLMDS-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
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None