Ignite Creation Date:
2024-05-06 @ 8:06 PM
Last Modification Date:
2024-10-26 @ 3:20 PM
Study NCT ID:
NCT06250595
Status:
RECRUITING
Last Update Posted:
2024-02-09
First Post:
2023-12-04
Brief Title:
European Rare Blood Disorders Platform ENROL
Sponsor:
Hospital Universitari Vall dHebron Research Institute
Organization:
Hospital Universitari Vall dHebron Research Institute
Study Overview
Official Title:
European Rare Blood Disorders Platform ENROL
Status:
RECRUITING
Status Verified Date:
2023-12
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
ENROL
Brief 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
ENROLs 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
ENROLs 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 EUnationallocal existing registries Data entry is also allowed with different levels of granularity to pursue ENROL aims including countsaggregated 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 HUVHVHIR ULBERASME 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 andor 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 ie 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
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 EUnationallocal existing registries Data entry is also allowed with different levels of granularity to pursue ENROL aims including countsaggregated 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 HUVHVHIR ULBERASME 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 andor 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 ie 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
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None