NCT04781790: RIME

French National Registry of Bone Marrow Failures

Recruiting now Last updated 4 March 2021

What this trial tests

trial testing Bone Marrow Failure in Bone Marrow Failure Syndrome in 5,000 participants. Currently enrolling.

Timeline

6 February 2017

Primary endpoint
6 February 2027

6 February 2027

Quick facts

Lead sponsor	Assistance Publique - Hôpitaux de Paris
Status	Recruiting now
Study type	OBSERVATIONAL
Enrollment	5,000
Start date	6 February 2017
Primary completion	6 February 2027
Estimated completion	6 February 2027
Sites	1 location across France

Drugs / interventions tested

• Bone Marrow Failure

Conditions studied

• Bone Marrow Failure Syndrome — all drugs for Bone Marrow Failure Syndrome →

Sponsor

Assistance Publique - Hôpitaux de Paris — full company profile →

Who can join

Eligibility, any sex, with Bone Marrow Failure Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Effectiveness of Iptacopan Versus C5 Inhibitors in Complement Inhibitor-Naive Patients With Paroxysmal Nocturnal Haemoglobinuria.
   Holt M, Kelly RJ, Fermont JM, Ansari S, et al · · 2025 · cited 1× · PMID 40395624 · DOI 10.1002/jha2.70055