INTRODUCING THE CMT&ME digital study,
the first international study of the impact
of cmt on patients’ daily life
CMT stands for Charcot-Marie-Tooth and is named after the three physicians who first described it in 1886: Jean-Martin Charcot and Pierre Marie from France; and Howard Henry Tooth from the UK.
IN PARTNERSHIP WITH
Charcot-Marie-Tooth disease (CMT) is a group of rare and inherited conditions that damage the peripheral nerves; it is also known as hereditary motor and sensory neuropathy (HMSN).
People with CMT may have muscle weakness in the feet, ankles, legs and hands, an awkward way of walking (gait), highly arched or very flat feet, numbness in the feet, arms and hands. The symptoms of CMT usually start to appear between the ages of five and 15, although they sometimes don't develop until well into middle age or later. CMT is a progressive condition caused by an inherited fault in one of the many genes responsible for the development of the peripheral nerves. This fault means the nerves become damaged over time. This means the symptoms slowly get worse, making everyday tasks increasingly difficult.
To date there’s been little research into the lives of people living with CMT. Pharnext, together with Experts and Patient Advocacy Associations from the international CMT community have decided to study the impact of the disease on people’s daily lives. With a desire to break ground for their researchers, Pharnext has committed to sponsor the two-year study in 6 countries including the US, UK, Germany, France, Italy and Spain. The aim of the study is to collect data on what it is like to live with CMT and to find out where and how treatment can improve quality of life and slow progression of the condition.
Tailoring of the digital platform and supporting materials has been informed by close collaboration with Scientific Experts, Patient Advocacy Organisations (PAOs) and patients. Working with PAOs in all six participating countries, the ethics-approved digital platform is available in five languages.
Data are collected in the real-world setting using “bring your own device” (BYOD) technology – participants use their own smartphone or tablet to complete questionnaires or surveys at their own convenience. Data are immediately submitted to a central data platform. Non-commercial researchers and Patient Advocacy Organisations (PAOs) can apply to the scientific advisory board for access to the anonymised aggregated data via dashboards in order to further knowledge of CMT and improve patient care.
What data are being collected?
The platform collects rich demographic and epidemiological data across all stages of disease, treatment, and beyond, including quality of life and symptom tracking. By taking part in the study and answering short surveys, you’ll help us gather invaluable insights into what it’s like to live with CMT.
Why digital data collection?
The advantages of electronic data collection are well documented and include reduced administrative burden for participants and administrators, the avoidance of errors in data collection and entry, and fewer items of missing data. Unlike paperwork, electronic data can be stored easily and securely and patients can complete questionnaires when and where they want to.
How do patients benefit?
Participants benefit from monitoring and recording how they feel, and the platform provides a convenient way for them to share their data with friends, family and healthcare professionals. Development of this platform has been collaborative.
At workshops with CMT patients, clinicians and PAO leaders, we canvassed future app users on the functionalities that they would appreciate having. Features such as a knowledge centre, with professionally curated content on CMT, and a symptom diary allowing patients to track how they are feeling at any time of day, have been added to the platform as a result of these collaborative sessions.
Who can access the data?
Applications to access the anonymized, aggregated dataset can be made to the study’s scientific advisory board. Approved non-commercial users will have free access to the research portal. The powerful online portal gives access to dashboards combining graphical representation of the data with appropriate statistical analysis, allowing data to be used readily in models and publications.
How to take part:
To install the app on an Android device or iOS, please click on the images below.
Once installed, the app will then ask a few questions to confirm the user’s eligibility, such as NHS number (or your country’s equivalent).
If the user is eligible, they will see more detailed information about the study, including “informed consent” questions that they will be asked to agree to.
The app icon will appear on the home screen of your device. Tap on the icon to begin. Note that you can install the app on more than one device and use the same login credentials on each device. You should also change the initial password you receive to one of your choosing.
For more information, you can also visit our FAQs page
For more information
Please contact: firstname.lastname@example.org