Myotubular and Centronuclear Myopathy Patient Registry_renewal
Research type
Research Database
IRAS ID
290690
Contact name
Chiara Marini-Bettolo
Contact email
Research summary
Myotubular and Centronuclear Myopathy Patient Registry (MTM and CNM Registry)
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
21/NE/0043
Date of REC Opinion
25 Feb 2021
REC opinion
Favourable Opinion
Data collection arrangements
This international registry will make the recruitment of MTM and CNM patients for clinical trials and studies easier. It will help 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 in which they might be interested. The best way to ensure that this happens is to collect patients’ details in a single, secure online database or “registry” that contains all the information that researchers will need, including details of each patient’s particular genetic mutation and other relevant information about their condition. A registry can also provide useful information on disease prevalence and the natural history (progression) of conditions. Patients affected by MTM or CNM 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 MTM and CNM Registry will have their details stored so that if they match the eligibility criteria of a clinical trial or study they can be made aware of it and have the chance to participate. This may result in patients receiving cutting edge treatments. In addition, registered patients will be kept informed about latest developments, including standards of care relevant to their disease, and will be provided with up to date information in the form of regular newsletters and email correspondence.
Research database title
Myotubular and Centronuclear Myopathy Patient Registry (MTM and CNM Registry)
Establishment organisation
Newcastle University
Establishment organisation address
John Walton Muscular Dystrophy Research Centre
International Centre for Life
Newcastle upon Tyne
NE1 3BZ