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. 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

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

37,090

participants targeted

Target at P75+ for all trials

Timeline

136mo left

Started Jul 2022

Longer than P75 for all trials

Geographic Reach

1 country

1 active site

Status

recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

15 years study duration

Study Progress26%

Jul 2022Jul 2037

Study Start

First participant enrolled

July 1, 2022

Completed

1.4 years until next milestone

First Submitted

Initial submission to the registry

December 4, 2023

Completed

2 months until next milestone

First Posted

Study publicly available on registry

February 9, 2024

Completed

13.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2037

Expected

Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2037

Last Updated

February 9, 2024

Status Verified

December 1, 2023

Enrollment Period

15 years

First QC Date

December 4, 2023

Last Update Submit

February 6, 2024

Conditions

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

Keywords

AnemiaBone Marrow FailureBleeding disorderIron metabolism disorderMyeloidLymphoidBlood cancerLeukemiaRed Cell membrane and Enzyme AbnormalitiesThalassemiaSickle Cell Disease

Outcome Measures

Primary Outcomes (1)

Demography and epidemiology
To collect and to describe demographics and epidemiological data of any type of RHDs.
15 years

Study Arms (6)

Inherited Rare Anaemia Disorders, including inherited Bone Marrow Failures

Patients with Inherited Rare Anemia Disorders, including inherited Bone Marrow Failures, stratified by gender, age, and/or variants/types if applicable.