RADeep Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs)

Sickle Cell Disease Clinical Trial

— RADeep  

Official title:

A Retrospective/Prospective, Multicenter European Epidemiological Platform for Patients Diagnosed With Rare Anemia Disorders (RADs) With Clinical Significance.

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06213402
• Other study ID #: PR(AMI)427/2021
• Status: Recruiting
• Start date: November 30, 2021
• Est. completion date: November 2036

Study information

• Verified date: December 2023
• Source: Hospital Universitari Vall d'Hebron Research Institute
• Contact: María del Mar Manú Pereira, PhD
• Phone: 0034934893000
• Email: mar.manu[@]vhir.org
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial Summary

Rare Anaemia Disorders (RADs) is a group of rare diseases characterized for presenting anaemia as the main clinical manifestation. Different medical entities classified as RADs by ORPHA classification are most of them chronic life threating disorders with many unmet needs for their proper clinical management creating an impact on European health systems. RADs present diagnostic challenges and their appropriate management requires from specialised multidisciplinary teams in Centers of expertise.

Although there are some examples of well-established national registries on RADs in EU, the lack of recommendations for Rare disease registries implementation and the lack of standards for interoperability has led to the fragmentation or unavailability of data on prevalence, survival, main clinical manifestations or treatments in most of the European countries.

Description:

The Rare Anaemia Disorders European Epidemiological Platform (RADeep) is an initiative endorsed by the European Reference Network on Rare Hematological Diseases (ERN-EuroBloodNet) under the frame of the European Blood Disorders Platform (ENROL), the ERN-EuroBloodNet umbrella platform officially endorsed by the European Hematology Association (EHA) for European patients' registries on rare haematological diseases. RADeep will share pseudonymised level data with ENROL.

RADeep supports the standardized collection of data of patients affected by any RADs at the European level, maximizing public benefit from data on RADs opened-up with the only restriction needed to guarantee patient rights and confidentiality, in agreement with the General Data Protection Regulation and applicable laws for cross-border sharing of personal data. RADeep has the following major objectives:

1. To collect and describe the demographics, disease-management, and treatment outcomes of patients diagnosed with RADs

2. To perform observational studies concerning research questions and to present outcomes in the fields of health related to organ damage and risk stratification for identification of trial cohorts for new drugs and/or development of research projects

3. To promote harmonization and best practices in the prevention, diagnosis, treatment and follow-up of RADs patients by the dissemination of reliable Guidelines and the translation of research results into clinical practice.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 32564
• Est. completion date: November 2036
• Est. primary completion date: December 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 0 Years to 100 Years

Eligibility

Inclusion Criteria:

• Patients must meet all of the following criteria to be included in the RADeep Registry

• Age from 0-100, both female and male

• Diagnosed as RADs (SCD, THAL, PKD, and other RADs THAL according to ORPHANET classification)

• Able and willing to provide written informed consent (patient or legal representative for minors)

Exclusion Criteria:

• Patient or legal representative for minors unwilling or unable to give consent

• Patients diagnosed with SCD or THAL (alpha-thalassaemia and beta-thalassaemia) traits or trait conditions for other recessive RADs

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Anemia, Hemolytic, Congenital
• Anemia, Sickle Cell
• Fanconi Anemia
• Fanconi Syndrome
• Iron Metabolism Disorders
• Sickle Cell Disease
• Thalassemia

Intervention

Other:
• Data collection from EHR.
Collection of clinical and laboratory data. Reviwe of the electronic health record

Locations

Country	Name	City	State
Spain	Vall d'hebron Research Institute - Vall d'Hebron Research Institute - University Hospital Vall d'Hebrón (VHIR/HUVH)	Barcelona	Catalunya

Sponsors (4)

Lead Sponsor	Collaborator
Hospital Universitari Vall d'Hebron Research Institute	Cyprus Institute of Neurology and Genetics, Erasme University Hospital, EuroBloodNet Association

Country where clinical trial is conducted

Spain, 

References & Publications (1)

Colombatti, R., Gutiérrez-Valle, V., Diot-Lefebvre, C., Labidi, I., Boaro, M.P., Tamana, S., Kountouris, P., Kleanthous, M., Gulbis,B., Mañú-Pereira, M. (2021, October 20). Rare Anaemia Disorders European Epidemiological Platform (RADeep). 17th Annual Sickle Cell & Thalassaemia Conference and 3rd Annual Academy Sickle Cell & Thalassaemia Conference (ASCAT 2022), London, United Kingdom of Great Britain and Northern Ireland.

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Estimation of Prevalence and Incidence of RADs	Demography and epidemiology; To collect and to describe demographics and epidemiological data of any type of RADs: Estimate the population frequency of each RAD disease group and disease survival; Estimate the diagnosis delay; Identify cohorts of patients for research/clinical trials; Estimate disease severity; Assess the use of specific treatments; Descriptive analyses will be undertaken at the end of the follow-up period using standard statistical methods to examine the subjects' demographics, disease characteristics and management. Data is updated yearly in an electronic CRF form while assuring homogenization in categorization and units.; Time-to-event analyses, namely Kaplan-Meier and Cox proportional hazard regression will be used to estimate overall survival.; Multivariate Cox proportional hazards regression models will be used to identify variables that are important to correlate survival.	15 years

External Links

•   EU EurobloodNet offficial Website
•   RADeep Official Website