Bone Marrow Failure SyndromeRIMENCT04781790
French National Registry of Bone Marrow Failures
An observational study of Bone Marrow Failure Syndrome, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Bone Marrow Failure Syndrome
- What is being tested?
- Bone Marrow Failure
- How many people
- 5,000 participants
- Where
- 1 siteFrance
- Study dates
- 6 Feb 2017to 6 Feb 2027
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with bone marrow failure syndromes (BMF)
Who may be able to join
- 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
Who cannot join
- With myelodysplastic syndrome occurring in a patient over the age of 50 in absence of genetical predispositions, familial forms and history of medullary hypoplasia
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
France1 site, 1 recruiting
- Recruiting
Hématologie Greffe
Paris
Regis PEFFAULT DE LATOUR
About this study
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.
- Conditions
- Bone Marrow Failure Syndrome
- Being tested
- Bone Marrow Failure
- Keywords
- aplastic anemia, Fanconi anemia, blackfan diamond anemia, telomeropathies, inherited aplastic anemia, acquired aplastic anemia, medullary hypoplasia
- Medical subject headings
- Bone Marrow Failure Disorders, Anemia, Aplastic, Fanconi Anemia, Anemia, Diamond-Blackfan
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Bone Marrow FailureOther
without interventional intervention . biological collection in the routine care Standard of care of patients with bone marrow failure.
| Study group | Role | What they receive |
|---|---|---|
| Patients with Bone marrow failure | Standard of care of patients with bone marrow failure Receives: Other: Bone Marrow Failure |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of bone marrow failure
Measured: at 10 years
Distribution of different bone marrow failure syndromes
Measured: at 10 years
Secondary outcomes (9)
Quality of life assessed by EORTC QLQ-C30- v3 questionnaire
Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.
EORTC QLQ-C30 Scoring Manual. Fayers PM et al. on behalf of the EORTC Quality of Life Group. EORTC, 2001. ISBN: 2-9300.
Measured: at inclusion
Quality of life assessed by EORTC QLQ-C30- v3 questionnaire
Quality of life evaluated using questionnaire "European Organization for Research and Treatment of Cancer Quality of Life Questionnaire" (EORTC QLQ-C30- v3). The QLQ-C30 is composed of both multi-item scales and single-item measures. These include five functional scales, three symptom scales, a global health status / QoL scale, and six single items. Each of the multi-item scales includes a different set of items - no item occurs in more than one scale. All of the scales and single-item measures range in score from 0 to 100. A high scale score represents a higher response level. A high score for a functional scale represents a high/healthy level of functioning. A high score for the global health status/ QoL represents a high QoL and a high score for a symptom scale/item represents a high level of symptomatology/problems.
EORTC QLQ-C30 Scoring Manual. Fayers PM et al. on behalf of the EORTC Quality of Life Group. EORTC, 2001. ISBN: 2-9300.
Measured: at 10 years
mechanisms underlying a clonal evolution or AML/MDS syndroms
Measured: at 10 years
Proportion of patients with new forms of constitutional aplasias
Measured: at 10 years
Proportion of patients with complications within each entity
Measured: at 10 years
Prognostic factor ans treatment response
Measured: at 10 years
Pathophysiology of bone marrow failures
Measured: at 10 years
Global response to treatment
Measured: at 10 years
Incidence and outcome of extra hematological complications including solid tumors, fertility
Measured: within 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2021
- Study start6 Feb 2017
- Primary completion6 Feb 2027 (estimated)
- Study completion6 Feb 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Bone Marrow Failure SyndromeStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 5,000 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04781790
- Sponsor's protocol ID
- NI17045J
- Acronym
- RIME
- Status last verified
- Feb 2021
- First submitted
- 1 Mar 2021
- Last updated
- 4 Mar 2021
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- French National Registry of Bone Marrow Failures: Prospective and Retrospective Database Associated to a Collection of Biological Samples: RIME Project
- ClinicalTrials.gov ID
- NCT04781790
- Acronym / sponsor ID
- RIME / NI17045J
- Conditions
- Bone Marrow Failure Syndrome
- Keywords
- aplastic anemia, Fanconi anemia, blackfan diamond anemia, telomeropathies, inherited aplastic anemia, acquired aplastic anemia, medullary hypoplasia
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 5,000 participants (estimated)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- 6 Feb 2017 (actual)
- Primary completion
- 6 Feb 2027 (estimated)
- Study completion
- 6 Feb 2027 (estimated)
- First posted
- 4 Mar 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with Bone marrow failure | – | Other: Bone Marrow FailureStandard of care of patients with bone marrow failure |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with bone marrow failure syndromes (BMF)
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (1 listed)
- With myelodysplastic syndrome occurring in a patient over the age of 50 in absence of genetical predispositions, familial forms and history of medullary hypoplasia
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Bone Marrow Failurewithout interventional intervention . biological collection in the routine care Standard of care of patients with bone marrow failure. | Other | Patients with Bone marrow failure |
Primary outcome
Prevalence of bone marrow failure
Time frame: at 10 years
Distribution of different bone marrow failure syndromes
Time frame: at 10 years
9 secondary outcomes
Quality of life assessed by EORTC QLQ-C30- v3 questionnaire
Time frame: at inclusion
Quality of life assessed by EORTC QLQ-C30- v3 questionnaire
Time frame: at 10 years
mechanisms underlying a clonal evolution or AML/MDS syndroms
Time frame: at 10 years
Proportion of patients with new forms of constitutional aplasias
Time frame: at 10 years
Proportion of patients with complications within each entity
Time frame: at 10 years
Prognostic factor ans treatment response
Time frame: at 10 years
Pathophysiology of bone marrow failures
Time frame: at 10 years
Global response to treatment
Time frame: at 10 years
Incidence and outcome of extra hematological complications including solid tumors, fertility
Time frame: within 10 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Bone Marrow Failure Syndrome, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Régis Peffault De Latour (+33142385073, regis.peffaultdelatour@aphp.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Prevalence of bone marrow failure (time frame: at 10 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Mar 2021.