Clinical Trial Details
— Status: Recruiting
Administrative data
| NCT number |
NCT03828279 |
| Other study ID # |
HAERegistry |
| Secondary ID |
|
| Status |
Recruiting |
| Phase |
|
| First received |
|
| Last updated |
|
| Start date |
October 1, 2017 |
| Est. completion date |
December 31, 2023 |
Study information
| Verified date |
January 2019 |
| Source |
HAE Global Registry Foundation |
| Contact |
marco cicardi, MD |
| Phone |
+39 0239042516 |
| Email |
marco.cicardi[@]unimi.it |
| Is FDA regulated |
No |
| Health authority |
|
| Study type |
Observational [Patient Registry]
|
Clinical Trial Summary
The objective of this international hereditary angioedema (HAE) register is to collect
homogeneous clinical and laboratory data on patients with HAE type I and II gathering better
information on the natural course of the disease and detecting therapeutic options to manage
it.
Description:
It is an international multicenter disease registry, the principal investigator at each
center is responsible for competent ethic committee/institutional review board approval and
for identifying co-investigators, who collect patients' informed consent and insert patients'
data. Participating patients own their data. Upon acceptance of the informed consent, the
treating physician transfers data of the case report form (CRF) to a specifically designed
electronic support (eCRF) and the patients enter data on attacks. Physicians at center
fill-in the eCRF with data for demography, diagnosis, prophylaxis and associated diseases.
Plasma levels of C1 inhibitor antigen and function and of C4 antigen document laboratory
diagnostic criteria. Each patient has a national identifier code. Patients fill-in attack
reports that encompass duration, severity and treatment of each angioedema episode. C1
inhibitor coding gene (SERPING1) genotype is entered according to the last Hugo nomenclature.
Yearly update of information is recommended, patients with follow up above two years are
moved in a separate area and excluded from analysis. Each patient prospectively fills data on
attacks. On a specific informed consent, the patient can agree on storing biological material
(plasma and nucleic acids) for research purposes.
The data processor is an innovative start-up (Cloud-R s.r.l. Milan, Italy) that manages all
data according to a specific contract and in compliance with current regulation on sensitive
data security and processing. The Registry, delivered in the mode of Registry-as-a-Service
(RaaS), is designed following General Data Protection Regulation (GDPR) guidelines, and
issues regular software and infrastructure enhancements as a part of the normal operational
mode. The system generates statistics of aggregated anonymized data following the
participating centers' hierarchy levels and Global Registry governance rules. Patients supply
data on attacks either on paper support or using a Web form or a mobile application. These
data will flow into a staging area for physician validation before being considered valid for
statistics. For each entry the system updates in real-time all the statistics and dashboards.
The platform has configurable functionalities to support data quality management. It provides
data format validation, integration to external qualified libraries, alerts, dashboards,
automatic index calculations, advanced filters and queries, data change log. This open
architecture, allows integration of the system, via standard API's, to external or
sub-registries, registries, biobanks and clinical bioinformatics tools, i.e. for specific
trial studies leveraging a specific cluster of patients already present in the Global
Registry.
An independent non-profit foundation (HGRF) made of representatives of patients' associations
is in charge for funding and delegates all the management to the HAE Global Registry Board
(HGRB). The HGRB is in charge for operational, assisted by the HAE Global Registry Scientific
Committee (HGRSC) for topics of competence. No registry member, center, group or board can
access the entire set of data. All registry members, as single or group, can propose studies
based on aggregated data by addressing the request to the HGRSC. Analysis and studies of data
at local centers can occur at any time. Members of HGRB and HGRSC are elected to be
representative of different cultural and geographic backgrounds. Their offices have a
two-year term with no more than one consecutive renewal. Angioedema centers can join the
Registry upon request to the HGRB . The registry quality control system periodically checks
Registry entries and compliance of eCRF with the source data. For each information, the
system will grant traceability of time and author.
