Bone Marrow Failure Syndrome Clinical Trial
Official title:
French National Registry of Bone Marrow Failures: Prospective and Retrospective Database Associated to a Collection of Biological Samples: RIME Project
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.
n/a
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Enrolling by invitation |
NCT02393508 -
The Impact of Red Cell Age on Product Utilization in the Chronically Transfused Outpatient Population
|
Phase 3 | |
| Terminated |
NCT01596699 -
Pilot Trial of Clofarabine Added to Standard Busulfan and Fludarabine for Conditioning Prior to Allogeneic Hematopoietic Cell Transplantation
|
Phase 2 | |
| Completed |
NCT00897260 -
Umbilical Cord Blood Transplantation As Treatment Of Adult Patients With Hematologic Disorders
|
N/A | |
| Active, not recruiting |
NCT01966367 -
CD34+ (Non-Malignant) Stem Cell Selection for Patients Receiving Allogeneic Stem Cell Transplantation
|
Phase 1/Phase 2 | |
| Recruiting |
NCT04819607 -
The Evaluating Multidisciplinary Bone Marrow Failure Care in Bone Marrow Failure and Related Disorders.
|
||
| Completed |
NCT03513328 -
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation
|
Phase 1/Phase 2 | |
| Recruiting |
NCT04356469 -
TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Non-Malignant Hematological Disorders in Children
|
Phase 2 | |
| Active, not recruiting |
NCT04558736 -
Haploidentical HCT for Severe Aplastic Anemia
|
Phase 2 | |
| Recruiting |
NCT01757145 -
Eltrombopag for Enhancing Platelet Engraftment in Adult Patients Undergoing Cord Blood Transplantation
|
Phase 2 | |
| Recruiting |
NCT02958462 -
Pre-myeloid Cancer and Bone Marrow Failure Clinic Study
|
N/A | |
| Recruiting |
NCT05236764 -
Haploidentical Hematopoietic Cell Transplantation Using TCR Alpha/Beta and CD19 Depletion
|
N/A | |
| Recruiting |
NCT04965597 -
Treosulfan-Based Conditioning Regimen Before a Blood or Bone Marrow Transplant for the Treatment of Bone Marrow Failure Diseases (BMT CTN 1904)
|
Phase 2 | |
| Available |
NCT03145545 -
Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC
|