Trial Title:
European Rare Blood Disorders Platform (ENROL)
NCT ID:
NCT06250595
Condition:
Anemia
Bone Marrow Failure
Bleeding Disorder
Iron Metabolism Disorders
Myeloma
Lymphoid Neoplasm
Myeloma, Malignant
Leukemia
Anemia, Sickle Cell
Thalassemia
Blood Cancer
Red Cell Membrane and Enzyme Abnormalities
Conditions: Official terms:
Multiple Myeloma
Neoplasms, Plasma Cell
Hematologic Neoplasms
Hemostatic Disorders
Anemia
Thalassemia
Blood Coagulation Disorders
Bone Marrow Failure Disorders
Pancytopenia
Anemia, Sickle Cell
Congenital Abnormalities
Metabolic Diseases
Iron Metabolism Disorders
Conditions: Keywords:
Anemia
Bone Marrow Failure
Bleeding disorder
Iron metabolism disorder
Myeloid
Lymphoid
Blood cancer
Leukemia
Red Cell membrane and Enzyme Abnormalities
Thalassemia
Sickle Cell Disease
Study type:
Observational [Patient Registry]
Overall status:
Recruiting
Study design:
Time perspective:
Other
Intervention:
Intervention type:
Other
Intervention name:
Collection of clinical and laboratory data from EHR.
Description:
Observational sutdy
Arm group label:
Acquired Bone Marrow Failures
Arm group label:
Hemochromatosis and other rare genetic disorders of iron metabolism and heme synthesis
Arm group label:
Inherited Rare Anaemia Disorders, including inherited Bone Marrow Failures
Arm group label:
Lymphoid malignancies
Arm group label:
Myeloid malignancies
Arm group label:
Rare bleeding-coagulation disorders and related diseases
Summary:
ENROL, the European Rare Blood Disorders Platform has been conceived in the core of
ERN-EuroBloodNet as an umbrella for both new and already existing registries on Rare
Hematological Diseases (RHDs). ENROL aims at avoiding fragmentation of data by promoting
the standards for patient registries' interoperability released by the EU RD platform.
ENROL's principle is to maximize public benefit from data on RHDs opened up through the
platform with the only restriction needed to guarantee patient rights and
confidentiality, in agreement with EU regulations for cross-border sharing of personal
data.
Accordingly, ENROL will map the EU-level demographics, survival rates, diagnosis methods,
genetic information, main clinical manifestations, and treatments in order to obtain
epidemiological figures and identify trial cohorts for basic and clinical research. To
this aim, ENROL will connect and facilitate the upgrading of existing RHD registries,
while promoting the building of new ones when / where lacking. Target-driven actions will
be carried out in collaboration with EURORDIS for educating patients and families about
the benefits of enrolment in such registries, including different cultural and linguistic
strategies.
The standardized collection and monitoring of disease-specific healthcare outcomes
through the ENROL user-friendly platform will determine how specialized care is
delivered, where are the gaps in diagnosis, care, or treatment and where best to allocate
financial, technical, or human resources.
Moreover, it will allow for promoting research, especially for those issues that remain
unanswered or sub-optimally addressed by the scientific community; furthermore, it will
allow promoting clinical trials for new drugs. ENROL will enable the generation of
evidence for better healthcare for RHD patients in the EU as the ultimate goal.
ENROL officially started on 1st June 2020 with a duration of 36 months. ENROL is
co-funded by the Health Programme of the European Union under the call for proposals
HP-PJ-2019 on Rare disease registries for the European Reference Networks. GA number
947670
Detailed description:
Study Title:
European Rare Blood Disorders Platform (ENROL)
Study Objectives:
The European Rare Blood Disorders Platform (ENROL) is conceived in the core of the
European Reference Network on Rare Hematological Diseases (ERN-EuroBloodNet), as the
umbrella platform for both new and already existing registries on Rare Hematological
Diseases (RHDs) avoiding fragmentation of data by promoting the interoperability
standards for patient registries.
ENROL aims at mapping demographics, diagnosis methods, genetic data, main clinical
manifestations and treatments at the EU level by enabling the connection, upgrading and
building of EU patients registries in line with EU data protection and interoperability
standards with the following major objectives:
Promoting research: allowing the identification of patient cohorts to facilitate the
development of collaborative projects on basic and clinical research on RHD
Epidemiological surveillance: pooling of available data on registries and databases
across European countries to monitor trends and provide essential epidemiologic
information on RHD while enabling the generation of evidence for the optimum allocation
of resources and health planning For this, ENROL will obtain comparable EU data for RHD
on demographics, survival rates, diagnosis methods, genetic information, main clinical
manifestations and treatments by promoting the interoperability standards for patient
registries in line with the European Platform on Rare Disease Registration (EU RD
Platform). ENROL is officially endorsed by the European Hematology Association (EHA).
Methodology:
Data on patients with RHDs will be collected retrospectively and prospectively at the
time of inclusion on the registry and at 12-month intervals for all registered patients.
The ENROL strategy for data gathering combines the exhaustiveness of data collection at
EU level for health planning and epidemiological purposes, with a higher level of RHD
data granularity for promoting research and identification of patients' cohorts.
Accordingly, the platform has been designed to integrate data from any available sources,
including Healthcare providers (ERN-EuroBloodNet members / Other EU healthcare providers)
and EU/national/local existing registries. Data entry is also allowed with different
levels of granularity to pursue ENROL aims, including counts/aggregated level data to
increase data exhaustiveness at the EU level required for the epidemiological
surveillance, and pseudonymised individual level data to promote research and facilitate
the identification of trial groups.
As defined in GDPR Art. 26, a Joint Controllership is being established where the
institutions conforming the Consortium (HUVH/VHIR, ULB/ERASME, CING and AP-HP) for the
jointly determine the purposes and means of processing and assume equal responsibilities
in terms of data protection.
Data processing and analyses will be conducted in various sub studies, after every 1000
patients included in the European Registry and/or at the end of each interim follow-up
period (every 12 months).
Disease coverage
The study population consist of both males and females aged from 0 to 100 year old
diagnosed as RHD according to ORPHANET classification (ORPHA 97992), including myeloid
and lymphoid tumors (ORPHA 68347), rare anaemia disorders (ORPHA 108997), rare
coagulation disorders (ORPHA 98429), and polycythemia (ORPHA 98427), and further
complemented with rare hereditary hemochromatosis (ORPHA220489), included in the disease
scope of ERN-EuroBloodNet following a request from well-established patient groups and
experts.
Accordingly, ENROL disease coverage results in more than 450 different entities with
differential clinical and etiological features i.e. oncological vs non-oncological,
hereditary vs acquired, or significant difference frequency, among others, which can be
classified into the following disease groups:
RAD: Inherited Rare Anaemia Disorders, including inherited Bone Marrow Failures BMF:
Acquired Bone Marrow Failures Bleeding: Rare bleeding-coagulation disorders and related
diseases HH-Iron: Hemochromatosis and other rare genetic disorders of iron metabolism and
heme synthesis Myeloid: Myeloid malignancies Lymphoid: Lymphoid malignancies
ENROL dataset has been conceived in a bottom-up design for ensuring the capture of the
common elements for rare diseases in line with the EU recommendations and with the EU RD
platform, and the key features common for the whole spectrum for RHDs. Furthermore, the
latest level can be stepped up for the definition of domain specific elements that
support the in-depth analysis.
In the spirit of ENROL aims to promote the connection and linkage of available data
sources and the creation of new registries where lacking. ENROL promotes collaborations
for supporting the a) upgrade the existing registries and b) create new European
registries in compliance with the standards of interoperability and ENROL policy.
Collaboration agreements will be in place for the transfer of a subset of the data
elements gathered to ENROL.
Study Duration and funding
An extensive recruitment period and follow-up, as well as geographical coverage, is
desirable for long-term sustainability of the European Epidemiological Platform.
Accordingly, ENROL has not an expected ending date but continue the pooling and
processing of data for an indeterminate period of time.
ENROL is co-funded by the Health Programme of the European Union under the call for
proposals HP-PJ-2019 on Rare disease registries for the European Reference Networks. GA
number 947670.
Criteria for eligibility:
Study pop:
Patients with Rare Hematological Disease
Sampling method:
Probability Sample
Criteria:
Inclusion Criteria:
- Patients must meet all of the following criteria to be included in the ENROL
Registry
- Age from 0-100, both female and male
- Diagnosed as RHDs according to ORPHANET classification
- Able and willing to provide written informed consent (patient or legal
representative for minors) if needed according to national legislation.
Exclusion Criteria:
- Patients diagnosed as traits or trait conditions for other recessive RHDs
Gender:
All
Minimum age:
N/A
Maximum age:
100 Years
Locations:
Facility:
Name:
María del Mar
Address:
City:
Barcelona
Zip:
08035
Country:
Spain
Status:
Recruiting
Contact:
Last name:
María del Mar Mañú Pereira, Biologist
Phone:
0034934893000
Email:
mar.manu@vhir.org
Contact backup:
Last name:
Victoria Gutiérrez Valle, Biotech
Phone:
+34934893000
Email:
victoria.gutierrez@vhir.org
Start date:
July 1, 2022
Completion date:
July 2037
Lead sponsor:
Agency:
Hospital Universitari Vall d'Hebron Research Institute
Agency class:
Other
Collaborator:
Agency:
ERN-EuroBloodNet (European Reference Network on Rare Hematological Diseases
Agency class:
Other
Source:
Hospital Universitari Vall d'Hebron Research Institute
Record processing date:
ClinicalTrials.gov processed this data on November 12, 2024
Source: ClinicalTrials.gov page:
https://clinicaltrials.gov/ct2/show/NCT06250595
https://eurobloodnet.eu/
https://health.ec.europa.eu/european-reference-networks/overview_en
https://eurobloodnet.eu/enrol-first/
