An observational study in Sickle Cell Disease, Thalassemia and Hemolytic; Anemia, Hereditary, Due to Enzyme Disorder, sponsored by Hospital Universitari Vall d'Hebron Research Institute. Recruiting at 1 site in Spain. Open to participants aged 0 Years to 100 Years. Per ClinicalTrials.gov, last updated 2024-01-19.
Sponsored by Hospital Universitari Vall d'Hebron Research Institute · Observational
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.
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:
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
This study's planned enrollment of 32,564 is above the median of 100 across 296 observational studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →Hospital Universitari Vall d'Hebron Research Institute is the lead sponsor of 276 studies on the registry; 64 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients with Rade Anemia Disease between 0 - 100 years old that accomplish all the inclusion criteria
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients with sickle cell disease and related diseases in current regular follow-ups in European-Union health centers
Other: Data collection from EHR.
Patients with Thalassemia disease and related diseases in current regular follow-ups in European-Union health centers, stratified by age, gender, and/or variants/type if applicable.
Other: Data collection from EHR.
Patients with Pyruvate Kinase Deficiency and related diseases in current regular follow-ups in European-Union health centers, stratified by age, gender, and/or variants/type if applicable.
Other: Data collection from EHR.
Patients with Reb Blood Cell membrane disorders and related diseases in current regular follow-ups in European-Union health centers, stratified by age, gender, and/or variants/type if applicable.
Other: Data collection from EHR.
Collection of clinical and laboratory data. Reviwe of the electronic health record
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.
Time frame: 15 years
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Started Nov 2021 (actual). Primary completion is estimated for Dec 2024, 37 months after the start.
Of 130 non-industry observational studies under Anemia, Sickle Cell, half reached primary completion within 48 months of starting (the median), counting those still running; a quarter within 23 and three quarters within 109. This study's planned 37 months is below that.
Counting every sponsor, half of 139 studies reached it within 49 months; a quarter within 24 and three quarters within 109.
How these figures are worked out, and which studies are left out of them.
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