Global FKRP Registry
Research type
Research Database
Full title
Global FKRP Registry
IRAS ID
136331
Contact name
Becky Davis
Contact email
Research summary
Global FKRP Registry
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
13/NE/0266
Date of REC Opinion
25 Nov 2013
REC opinion
Favourable Opinion
Data collection arrangements
This international registry will make the recruitment of FKRP patients for trials/studies much easier by helping to identify suitable patients for particular trials/studies more readily and enable them to be contacted and informed more quickly when there is a trial/study they might be interested in. The best way to ensure that this happens is to make sure that patients’ details are all collected in a single database or “registry” that contains all the information that researchers will need, including each patient’s particular genetic defect and other key information about that condition. A registry can also provide useful information on disease prevalence and the natural history (progression) of conditions. Patients affected by conditions caused by mutations in the Fukutin Related Protein (FKRP) gene are very rare. Without a patient registry to gather details from all patients worldwide in one place, finding enough patients for a meaningful trial can take years, delaying the testing of potential therapies. Patient numbers in different countries will be very low due to the rare nature of these conditions so it is more efficient to have one international registry that collects the information rather than having many national registries.
Research programme
Patients registered with the Global FKRP Registry will have their details stored so that if they match the eligibility criteria of a clinical trial/study they can be made aware of this and have the chance of participating. This may result in patients receiving cutting edge treatments. Secondly, registered patients will also be kept informed about latest developments, including standards of care relevant to their disease, and will also be provided with up to date information in the form of annual newsletters and email correspondence.
Research database title
Global FKRP Registry
Establishment organisation
Newcastle University
Establishment organisation address
Institute of Genetic Medicine
International Centre for Life
NE1 3BZ