Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT06250595 |
| Other study ID # |
PR(AMI)294/2022 |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
July 1, 2022 |
| Est. completion date |
July 2037 |
Study information
| Verified date |
December 2023 |
| Source |
Hospital Universitari Vall d'Hebron Research Institute |
| Contact |
María del Mar Manú Pereira, PhD |
| Phone |
+34934893000 |
| Email |
mar.manu[@]vhir.org |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational [Patient Registry]
|
Clinical Trial 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
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.
