A patient registry collects and stores patient medical information, family history and other related information in a safe anonymised way for use in clinical research.
The purpose of the MLD registry is to collect and store clinical and other information from individuals with the same or related disease in the UK to get a better understanding as to how affected individuals progress.
Patient information in this registry will be used to create a natural history of MLD types for clinical research and experimental clinical trials to better understand rare diseases and to develop new treatments. Researchers studying MLD need accurate information to understand how the disease affects people.
In addition, researchers may access the MLD registry to locate people who may be eligible to participate in particular studies, such as studies to test new treatments. All requests for research will be carefully vetted and only anonymised data released.
The registry is hosted with OpenApp, a company based in Ireland with an excellent background in supporting patient registries in many rare diseases.
MLD Support Association UK and several families have created the data fields to ensure that we collect pertinent information. Currently we are reviewing all our documentation to ensure that all the information sheets, consent forms etc. fulfil all regulatory requirements for confidentiality, security and data protection.
Once the Registry is live, families will be provided with all the information to be able to make an informed decision on participation. The patient/parent/carer will be asked to sign an informed consent form which will be retained in a safe environment. This consent can be withdrawn at any time. Members of MLD Support Association UK will be available to answer any questions.
If families decide to join the Registry, the questionnaires will be available on paper and electronically and once completed will go to Jackie Imrie to be entered into the data base. After the data is entered the original documents will be stored in a safe environment or destroyed depending on data handling guidelines.