Platform for Rare Blood Disorders in Europe
European Rare Blood Disorders Platform (ENROL)
This study is trying to gather and share information about rare blood disorders across Europe to help researchers understand these conditions better and improve treatment options for patients.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 37090 (estimated) |
| Ages | N/A to 100 Years |
| Sex | All |
| Sponsor | Hospital Universitari Vall d'Hebron Research Institute Academic / other |
| Locations | 1 site (Barcelona, Catalonia) |
| Trial ID | NCT06250595 on ClinicalTrials.gov |
What this trial studies
The European Rare Blood Disorders Platform (ENROL) aims to consolidate data from various registries on Rare Hematological Diseases (RHDs) to enhance research and epidemiological surveillance. By promoting interoperability standards for patient registries, ENROL will map demographics, survival rates, diagnosis methods, genetic information, clinical manifestations, and treatments across Europe. This observational study will facilitate the identification of patient cohorts for collaborative research projects while ensuring compliance with EU data protection regulations. The platform seeks to maximize public benefit from RHD data while safeguarding patient rights and confidentiality.
Who should consider this trial
Good fit: Ideal candidates include individuals aged 0-100 diagnosed with Rare Hematological Diseases as per ORPHANET classification.
Not a fit: Patients diagnosed only with traits or trait conditions for other recessive RHDs may not benefit from this study.
Why it matters
Potential benefit: If successful, this platform could significantly improve the understanding and treatment of rare blood disorders across Europe.
How similar studies have performed: Other studies have shown success in utilizing registry data for rare diseases, making this approach promising yet still innovative.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients must meet all of the following criteria to be included in the ENROL Registry * Age from 0-100, both female and male * Diagnosed as RHDs according to ORPHANET classification * Able and willing to provide written informed consent (patient or legal representative for minors) if needed according to national legislation. Exclusion Criteria: * Patients diagnosed as traits or trait conditions for other recessive RHDs
Where this trial is running
Barcelona, Catalonia
- María del Mar — Barcelona, Catalonia, Spain (Recruiting)
Study contacts
- Principal investigator: María del Mar Manú Pereira, PhD — Vall d'Hebron Institut de Recerca / University Hospital Vall d'Hebron (VHIR / HUVH)
- Study coordinator: María del Mar Manú Pereira, PhD
- Email: mar.manu@vhir.org
- Phone: +34934893000
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.